JP2017535266A - 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 - Google Patents
筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 Download PDFInfo
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- JP2017535266A JP2017535266A JP2017525847A JP2017525847A JP2017535266A JP 2017535266 A JP2017535266 A JP 2017535266A JP 2017525847 A JP2017525847 A JP 2017525847A JP 2017525847 A JP2017525847 A JP 2017525847A JP 2017535266 A JP2017535266 A JP 2017535266A
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- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2330/00—Production
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- C12N2330/51—Specially adapted vectors
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- General Chemical & Material Sciences (AREA)
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- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pain & Pain Management (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022030717A JP2022081546A (ja) | 2014-11-14 | 2022-03-01 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
| JP2024050822A JP2024079794A (ja) | 2014-11-14 | 2024-03-27 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462079588P | 2014-11-14 | 2014-11-14 | |
| US62/079,588 | 2014-11-14 | ||
| US201562211992P | 2015-08-31 | 2015-08-31 | |
| US62/211,992 | 2015-08-31 | ||
| US201562234466P | 2015-09-29 | 2015-09-29 | |
| US62/234,466 | 2015-09-29 | ||
| PCT/US2015/060562 WO2016077687A1 (en) | 2014-11-14 | 2015-11-13 | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020159246A Division JP2021007394A (ja) | 2014-11-14 | 2020-09-24 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
| JP2022030717A Division JP2022081546A (ja) | 2014-11-14 | 2022-03-01 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017535266A true JP2017535266A (ja) | 2017-11-30 |
| JP2017535266A5 JP2017535266A5 (2) | 2018-10-25 |
Family
ID=55955103
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017525847A Pending JP2017535266A (ja) | 2014-11-14 | 2015-11-13 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
| JP2020159246A Pending JP2021007394A (ja) | 2014-11-14 | 2020-09-24 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
| JP2022030717A Pending JP2022081546A (ja) | 2014-11-14 | 2022-03-01 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
| JP2024050822A Pending JP2024079794A (ja) | 2014-11-14 | 2024-03-27 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020159246A Pending JP2021007394A (ja) | 2014-11-14 | 2020-09-24 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
| JP2022030717A Pending JP2022081546A (ja) | 2014-11-14 | 2022-03-01 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
| JP2024050822A Pending JP2024079794A (ja) | 2014-11-14 | 2024-03-27 | 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法 |
Country Status (14)
| Country | Link |
|---|---|
| US (5) | US10597660B2 (2) |
| EP (1) | EP3218484A4 (2) |
| JP (4) | JP2017535266A (2) |
| KR (2) | KR20230169197A (2) |
| CN (2) | CN107109407A (2) |
| AU (2) | AU2015346162B2 (2) |
| CA (2) | CA3193811A1 (2) |
| HK (1) | HK1244299A1 (2) |
| IL (3) | IL292999A (2) |
| MX (2) | MX2017006216A (2) |
| RU (2) | RU2716422C2 (2) |
| SG (1) | SG11201703281RA (2) |
| WO (1) | WO2016077687A1 (2) |
| ZA (1) | ZA201702991B (2) |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019132624A1 (ko) * | 2017-12-29 | 2019-07-04 | 주식회사 헬릭스미스 | 하이브리드 hgf 유전자가 도입된 aav(아데노-연관 바이러스) 벡터 |
| KR20210131992A (ko) * | 2018-12-14 | 2021-11-03 | 바이오젠 엠에이 인코포레이티드 | 근위축 측삭 경화증의 치료 및 방지를 위한 조성물 및 방법 |
| JP2021529513A (ja) * | 2018-07-02 | 2021-11-04 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症および脊髄に関連する障害の治療 |
| JP2023537980A (ja) * | 2020-08-12 | 2023-09-06 | ユーシービー バイオファルマ エスアールエル | メチルｃｐｇ結合タンパク質２（ｍｅｃｐ２）プロモータ配列を含む核酸構築体を用いた遺伝子治療 |
| US12116589B2 (en) | 2017-10-16 | 2024-10-15 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RS60707B1 (sr) | 2014-04-01 | 2020-09-30 | Biogen Ma Inc | Kompozicije za modulaciju ekspresije sod-1 |
| CN119876138A (zh) | 2014-11-14 | 2025-04-25 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CA3024449A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| GB201609597D0 (en) * | 2016-06-01 | 2016-07-13 | Univ Sheffield | Therapy |
| JP2020518259A (ja) * | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| EP3618839A4 (en) * | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| JP2020518266A (ja) * | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 調節性ポリヌクレオチド |
| WO2019079242A1 (en) * | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| AU2018354195A1 (en) * | 2017-10-23 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| EP3785279A4 (en) * | 2018-05-31 | 2022-07-27 | Immunity Pharma Ltd. | COMPOSITIONS AND METHODS OF USE THEREOF IN THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US12060567B2 (en) | 2018-06-13 | 2024-08-13 | Voyager Therapeutics, Inc. | Engineered untranslated regions (UTR) for AAV production |
| WO2020010035A1 (en) | 2018-07-02 | 2020-01-09 | Voyager Therapeutics, Inc. | Cannula system |
| AU2019310459A1 (en) | 2018-07-24 | 2021-02-18 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
| WO2020072849A1 (en) | 2018-10-04 | 2020-04-09 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| WO2020072844A1 (en) | 2018-10-05 | 2020-04-09 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| CA3115810A1 (en) | 2018-10-11 | 2020-04-16 | Decibel Therapeutics, Inc. | Aav1 vectors and uses thereof for treatment of otic indications |
| CA3116701A1 (en) | 2018-10-15 | 2020-04-23 | Voyager Therapeutics, Inc. | Expression vectors for large-scale production of raav in the baculovirus/sf9 system |
| MA54383A (fr) * | 2018-12-06 | 2021-10-13 | Biogen Ma Inc | Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique |
| JP2022522995A (ja) | 2019-01-18 | 2022-04-21 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖ粒子を生成するための方法及びシステム |
| KR20210130158A (ko) | 2019-01-31 | 2021-10-29 | 오레곤 헬스 앤드 사이언스 유니버시티 | Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법 |
| EP3962536A1 (en) | 2019-04-29 | 2022-03-09 | Voyager Therapeutics, Inc. | Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors |
| WO2020223296A1 (en) * | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| WO2021030125A1 (en) | 2019-08-09 | 2021-02-18 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| TW202122582A (zh) | 2019-08-26 | 2021-06-16 | 美商航海家醫療公司 | 病毒蛋白之控制表現 |
| US20220389067A1 (en) * | 2019-11-05 | 2022-12-08 | The Board Of Trustees Of The Leland Stanford Junior University | Methods of Treating Neurodegenerative Diseases Caused by G4C2 Expansion in C9ORF72 |
| EP3892283A1 (en) * | 2020-04-09 | 2021-10-13 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Nucleic acids encoding human fus protein and use in the treatment of amyotrophic lateral sclerosis (als) |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| WO2022032153A1 (en) | 2020-08-06 | 2022-02-10 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| WO2022109368A1 (en) * | 2020-11-20 | 2022-05-27 | Texas Tech University System | Methods of treating or preventing neurological disorders using zpr1 |
| WO2022174000A2 (en) * | 2021-02-12 | 2022-08-18 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| US20240141377A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
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| CN116270704A (zh) * | 2023-02-24 | 2023-06-23 | 西交利物浦大学 | 一种抑制sod1基因表达的药物组合物及其应用 |
| WO2024226761A2 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| IL324720A (en) * | 2023-05-26 | 2026-01-01 | Adarx Pharmaceuticals Inc | SOD1 modulators and methods of using them |
| CN116832050B (zh) * | 2023-07-06 | 2026-04-24 | 南京大学 | 一种用于治疗肌萎缩侧索硬化症的rna递送系统 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007109097A2 (en) * | 2006-03-16 | 2007-09-27 | Alnylam Pharmaceuticals, Inc. | RNAi MODULATION OF TGF-BETA AND THERAPEUTIC USES THEREOF |
| JP2013143917A (ja) * | 2012-01-13 | 2013-07-25 | Mie Univ | 線維症予防又は治療剤 |
Family Cites Families (523)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE68923613T2 (de) | 1988-06-14 | 1996-01-04 | Chiron Corp | Superoxid-dismutaseanaloge mit neuen bindeeigenschaften. |
| US5171680A (en) | 1988-06-14 | 1992-12-15 | Chiron Corporation | Superoxide dismutase analogs having novel binding properties |
| FR2640638B1 (fr) | 1988-12-20 | 1991-02-15 | Commissariat Energie Atomique | Bioreacteur et dispositif pour la culture de cellules animales |
| WO1991018088A1 (en) | 1990-05-23 | 1991-11-28 | The United States Of America, Represented By The Secretary, United States Department Of Commerce | Adeno-associated virus (aav)-based eucaryotic vectors |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5252479A (en) | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| AU680459B2 (en) | 1992-12-03 | 1997-07-31 | Genzyme Corporation | Gene therapy for cystic fibrosis |
| FR2705361B1 (fr) | 1993-05-18 | 1995-08-04 | Centre Nat Rech Scient | Vecteurs viraux et utilisation en thérapie génique. |
| FR2705686B1 (fr) | 1993-05-28 | 1995-08-18 | Transgene Sa | Nouveaux adénovirus défectifs et lignées de complémentation correspondantes. |
| BR9405507A (pt) | 1993-07-13 | 1999-05-25 | Rhone Poulenc Rorer Sa | Adenovirus recombinante defeituoso linhagem celular e composição farmaceutica |
| US5693531A (en) | 1993-11-24 | 1997-12-02 | The United States Of America As Represented By The Department Of Health And Human Services | Vector systems for the generation of adeno-associated virus particles |
| CA2187626C (en) | 1994-04-13 | 2009-11-03 | Michael G. Kaplitt | Aav-mediated delivery of dna to cells of the nervous system |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US20020159979A1 (en) | 1994-06-06 | 2002-10-31 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| US5625048A (en) | 1994-11-10 | 1997-04-29 | The Regents Of The University Of California | Modified green fluorescent proteins |
| IL116816A (en) | 1995-01-20 | 2003-05-29 | Rhone Poulenc Rorer Sa | Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof |
| US5652224A (en) | 1995-02-24 | 1997-07-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism |
| US5741657A (en) | 1995-03-20 | 1998-04-21 | The Regents Of The University Of California | Fluorogenic substrates for β-lactamase and methods of use |
| US5756283A (en) | 1995-06-05 | 1998-05-26 | The Trustees Of The University Of Pennsylvania | Method for improved production of recombinant adeno-associated viruses for gene therapy |
| US6281010B1 (en) | 1995-06-05 | 2001-08-28 | The Trustees Of The University Of Pennsylvania | Adenovirus gene therapy vehicle and cell line |
| US5688676A (en) | 1995-06-07 | 1997-11-18 | Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6197293B1 (en) | 1997-03-03 | 2001-03-06 | Calydon, Inc. | Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof |
| US6676935B2 (en) | 1995-06-27 | 2004-01-13 | Cell Genesys, Inc. | Tissue specific adenoviral vectors |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| US6265389B1 (en) | 1995-08-31 | 2001-07-24 | Alkermes Controlled Therapeutics, Inc. | Microencapsulation and sustained release of oligonucleotides |
| US5858351A (en) | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
| US5962313A (en) | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
| US5846528A (en) | 1996-01-18 | 1998-12-08 | Avigen, Inc. | Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence |
| US5952221A (en) | 1996-03-06 | 1999-09-14 | Avigen, Inc. | Adeno-associated virus vectors comprising a first and second nucleic acid sequence |
| EP1736554B1 (en) | 1996-05-29 | 2013-10-09 | Cornell Research Foundation, Inc. | Detection of nucleic acid sequence differences using coupled ligase detection and polymerase chain reactions |
| US7026468B2 (en) | 1996-07-19 | 2006-04-11 | Valentis, Inc. | Process and equipment for plasmid purification |
| US20020037867A1 (en) | 1999-02-26 | 2002-03-28 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| AU722624B2 (en) | 1996-09-06 | 2000-08-10 | Trustees Of The University Of Pennsylvania, The | An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| US5866552A (en) | 1996-09-06 | 1999-02-02 | The Trustees Of The University Of Pennsylvania | Method for expressing a gene in the absence of an immune response |
| WO1998010087A1 (en) | 1996-09-06 | 1998-03-12 | Trustees Of The University Of Pennsylvania | Chimpanzee adenovirus vectors |
| IL128736A0 (en) | 1996-09-06 | 2000-01-31 | Univ Pennsylvania | Methods using cre-lox for production of recombinant adeno-associated viruses |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US7732129B1 (en) | 1998-12-01 | 2010-06-08 | Crucell Holland B.V. | Method for the production and purification of adenoviral vectors |
| ES2278399T3 (es) | 1996-11-20 | 2007-08-01 | Introgen Therapeutics, Inc. | Metodo mejorado para la produccion y purificacion de vectores adenovirales. |
| AU741605B2 (en) | 1996-12-18 | 2001-12-06 | Targeted Genetics Corporation | AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6710036B2 (en) | 1997-07-25 | 2004-03-23 | Avigen, Inc. | Induction of immune response to antigens expressed by recombinant adeno-associated virus |
| US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| CA2304131A1 (en) | 1997-09-19 | 1999-04-01 | James M. Wilson | Method for gene transfer using bcl2 and compositions useful therein |
| CA2303768C (en) | 1997-09-19 | 2009-11-24 | The Trustees Of The University Of Pennsylvania | Methods and vector constructs useful for production of recombinant aav |
| CA2304168A1 (en) | 1997-09-19 | 1999-04-01 | The Trustees Of The University Of Pennsylvania | Methods and cell line useful for production of recombinant adeno-associated viruses |
| US6642051B1 (en) | 1997-10-21 | 2003-11-04 | Targeted Genetics Corporation | Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors |
| IT1297074B1 (it) | 1997-11-21 | 1999-08-03 | Angeletti P Ist Richerche Bio | Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le |
| JP2001526900A (ja) | 1997-12-23 | 2001-12-25 | イントロヘーネ ベスローテン フェンノートシャップ | 標的細胞の染色体ｄｎａへの外来遺伝子情報の組み込みに有用な、アデノ随伴ウイルスおよびアデノウイルスのキメラ組換えウイルス |
| US6410300B1 (en) | 1998-01-12 | 2002-06-25 | The University Of North Carolina At Chapel Hill | Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV |
| WO1999043360A1 (en) | 1998-02-26 | 1999-09-02 | The Trustees Of The University Of Pennsylvania | Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex |
| US6953690B1 (en) | 1998-03-20 | 2005-10-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US6521426B1 (en) | 1998-04-08 | 2003-02-18 | Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. | Preparation of recombinant adenovirus carrying a rep gene of adeno-associated virus |
| FR2778413B1 (fr) | 1998-05-07 | 2000-08-04 | Immunotech Sa | Nouveaux reactifs et methode de lyse des erythrocytes |
| EP1078096A1 (en) | 1998-05-11 | 2001-02-28 | Ariad Gene Therapeutics, Inc. | Multiviral compositions and uses thereof |
| EP1849872A1 (en) | 1998-05-20 | 2007-10-31 | University Of Iowa Research Foundation | Adeno-associated virus vectors and uses thereof |
| US6436392B1 (en) | 1998-05-20 | 2002-08-20 | University Of Iowa Research Foundation | Adeno-associated virus vectors |
| EP1082445A2 (en) | 1998-05-27 | 2001-03-14 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated expression of factor viii activity |
| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
| EP1082413B1 (en) | 1998-05-28 | 2008-07-23 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav5 vector and uses thereof |
| US6984517B1 (en) | 1998-05-28 | 2006-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector and uses thereof |
| GB2338236B (en) | 1998-06-13 | 2003-04-09 | Aea Technology Plc | Microbiological cell processing |
| US6900049B2 (en) | 1998-09-10 | 2005-05-31 | Cell Genesys, Inc. | Adenovirus vectors containing cell status-specific response elements and methods of use thereof |
| WO2000022152A1 (en) | 1998-10-13 | 2000-04-20 | Avigen, Inc. | Compositions and methods for producing recombinant adeno-associated virus |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| AU764130B2 (en) | 1998-10-27 | 2003-08-14 | Crucell Holland B.V. | Improved AAV vector production |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| US6689600B1 (en) | 1998-11-16 | 2004-02-10 | Introgen Therapeutics, Inc. | Formulation of adenovirus for gene therapy |
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| US6225113B1 (en) | 1998-12-04 | 2001-05-01 | Genvec, Inc. | Use of trans-activation and cis-activation to modulate the persistence of expression of a transgene in an at least E4-deficient adenovirus |
| US6387368B1 (en) | 1999-02-08 | 2002-05-14 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| DE19905501B4 (de) | 1999-02-10 | 2005-05-19 | MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie | Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels |
| US6509150B1 (en) | 1999-03-05 | 2003-01-21 | Universite De Nantes | Compositions and methods for recombinant Adeno-Associated Virus production |
| JP4693244B2 (ja) | 1999-03-18 | 2011-06-01 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法 |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| JP2002542805A (ja) | 1999-04-30 | 2002-12-17 | ユニバーシティ オブ フロリダ | アデノ随伴ウイルス送達リボザイム組成物および使用方法 |
| WO2000075353A1 (en) | 1999-06-02 | 2000-12-14 | Trustees Of The University Of Pennsylvania | Compositions and methods useful for production of recombinant viruses which require helper viruses |
| JP4969002B2 (ja) | 1999-06-08 | 2012-07-04 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | ｒＡＡＶ形質導入を増加するための化合物および方法 |
| CA2382483A1 (en) | 1999-08-20 | 2001-03-01 | Johns Hopkins University School Of Medicine | Methods and compositions for the construction and use of fusion libraries |
| US6365394B1 (en) | 1999-09-29 | 2002-04-02 | The Trustees Of The University Of Pennsylvania | Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus |
| AU1071701A (en) | 1999-09-29 | 2001-04-30 | Trustees Of The University Of Pennsylvania, The | Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor |
| US7241447B1 (en) | 1999-10-07 | 2007-07-10 | University Of Iowa Research Foundation | Adeno-associated virus vectors and uses thereof |
| EP1224313A1 (en) | 1999-10-07 | 2002-07-24 | University of Iowa Research Foundation | Adeno-associated viruses and uses thereof |
| AU1436401A (en) | 1999-10-21 | 2001-05-14 | Board Of Trustees Of The University Of Arkansas, The | RepO-associated virus AAV REP78 major regulatory protein, mutants thereof and uses thereof |
| WO2001036623A2 (en) | 1999-11-05 | 2001-05-25 | Avigen, Inc. | Ecdysone-inducible adeno-associated virus expression vectors |
| AU1775901A (en) | 1999-11-17 | 2001-05-30 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| AU1954001A (en) | 1999-12-10 | 2001-06-18 | Ariad Gene Therapeutics, Inc. | Methods for high level expression of genes in primates |
| US7638120B2 (en) | 2000-03-14 | 2009-12-29 | Thomas Jefferson University | High transgene expression of a pseudotyped adeno-associated virus type |
| CA2373110A1 (en) | 2000-03-14 | 2001-09-20 | Neurologix, Inc. | Production of chimeric capsid vectors |
| US6468524B1 (en) | 2000-03-22 | 2002-10-22 | The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services | AAV4 vector and uses thereof |
| US6855314B1 (en) | 2000-03-22 | 2005-02-15 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector for transducing brain cells and lung cells |
| US7048920B2 (en) | 2000-03-24 | 2006-05-23 | Cell Genesys, Inc. | Recombinant oncolytic adenovirus for human melanoma |
| PT1309726E (pt) | 2000-03-30 | 2010-03-08 | Whitehead Biomedical Inst | Mediadores de interferência por rna específicos de sequência de rna |
| GB0009887D0 (en) | 2000-04-20 | 2000-06-07 | Btg Int Ltd | Cytotoxic agents |
| US20030013189A1 (en) | 2000-04-28 | 2003-01-16 | Wilson James M. | Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream |
| AU2001257611A1 (en) | 2000-04-28 | 2001-11-12 | Avigen, Inc. | Polynucleotides for use in recombinant adeno-associated virus virion production |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| NZ522840A (en) | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
| US20020106381A1 (en) | 2000-06-13 | 2002-08-08 | High Katherine A. | Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus |
| ATE364698T1 (de) | 2000-07-18 | 2007-07-15 | Takeda Pharmaceutical | Neues, physiologisch aktives peptid und dessen verwendung |
| US6329181B1 (en) | 2000-08-07 | 2001-12-11 | Neurologix, Inc. | Helper functions for recombinant vector production |
| US6593123B1 (en) | 2000-08-07 | 2003-07-15 | Avigen, Inc. | Large-scale recombinant adeno-associated virus (rAAV) production and purification |
| CA2419245A1 (en) | 2000-08-17 | 2002-02-21 | Keiya Ozawa | Adeno-associated virus-mediated delivery of angiogenic factors |
| DE10044384A1 (de) | 2000-09-08 | 2002-04-18 | Medigene Ag | Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung |
| FR2813891B1 (fr) | 2000-09-14 | 2005-01-14 | Immunotech Sa | Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications |
| WO2002034264A1 (en) | 2000-10-24 | 2002-05-02 | Mitsubishi Pharma Corporation | Remedies for amyotrophic lateral sclerosis (als) |
| JP2002153278A (ja) | 2000-11-22 | 2002-05-28 | Hisamitsu Pharmaceut Co Inc | ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法 |
| WO2002070719A2 (en) | 2001-01-19 | 2002-09-12 | Trustees Of The University Of Pennsylvania | Regulatable gene expression system |
| WO2002071843A1 (en) | 2001-03-14 | 2002-09-19 | Genteric, Inc. | Recombinant adeno-assocaited virus-mediated gene transfer via retroductal infusion of virions |
| CA2790034A1 (en) | 2001-06-21 | 2003-01-03 | Isis Pharmaceuticals, Inc. | Antisense modulation of superoxide dismutase 1, soluble expression |
| US20040136963A1 (en) | 2001-06-22 | 2004-07-15 | The Trustees Of The University Of Pennsylvania | Simian adenovirus vectors and methods of use |
| ES2375557T3 (es) | 2001-06-22 | 2012-03-02 | The Trustees Of The University Of Pennsylvania | Adenovirus recombinantes que comprenden prote�?nas de adenovirus de simios y usos de los mismos. |
| AU2007201330C1 (en) | 2001-07-12 | 2011-06-09 | University Of Massachusetts | In vivo production of small interfering RNAs that mediate gene silencing |
| EP2345720A3 (en) | 2001-07-12 | 2012-01-25 | University of Massachusetts | In vivo production of small interfering RNAs that mediate gene silencing |
| EP1900815B1 (en) | 2001-07-12 | 2016-09-07 | University of Massachusetts | In vivo production of small interfering RNAs that mediate gene silencing |
| US8241622B2 (en) | 2001-07-13 | 2012-08-14 | University Of Iowa Research Foundation | Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences |
| US20030016777A1 (en) | 2001-07-18 | 2003-01-23 | Alain Vandergheynst | TIG welded MOX fuel rod |
| EP1279740A1 (en) | 2001-07-26 | 2003-01-29 | Vrije Universiteit Brussel | Recombinant vector derived from adeno-associated virus for gene therapy |
| JP2004538005A (ja) | 2001-08-08 | 2004-12-24 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | シアル酸に結合するタンパク質を有するウイルスベクターの精製法 |
| US20030092161A1 (en) | 2001-09-19 | 2003-05-15 | The Trustees Of The University Of Pennsylvania | Compositions and methods for production of recombinant viruses, and uses therefor |
| EP2390331B1 (en) | 2001-09-28 | 2018-04-25 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | MicroRNA molecules |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| WO2003042361A2 (en) | 2001-11-09 | 2003-05-22 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| EP1453543B1 (en) | 2001-11-21 | 2017-08-30 | The Trustees Of The University Of Pennsylvania | Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use |
| EP1944043A1 (en) | 2001-11-21 | 2008-07-16 | The Trustees of the University of Pennsylvania | Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use |
| EP1453536A4 (en) | 2001-12-12 | 2009-08-26 | Mayne Pharma Int Pty Ltd | COMPOSITION FOR PRESERVING VIRUSES |
| AU2002359284A1 (en) | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| WO2003053476A1 (en) | 2001-12-19 | 2003-07-03 | Lijun Wang | Adeno-associated virus-mediated delivery of gdnf to skeletal muscles |
| JP2005538929A (ja) | 2002-01-16 | 2005-12-22 | ダイナル バイオテック エイエスエイ | 単一サンプルからの核酸及びタンパク質の単離方法 |
| US20030180756A1 (en) * | 2002-03-21 | 2003-09-25 | Yang Shi | Compositions and methods for suppressing eukaryotic gene expression |
| GB0208390D0 (en) | 2002-04-11 | 2002-05-22 | Univ London | Adeno-associated virus producer system |
| US20030198620A1 (en) | 2002-04-16 | 2003-10-23 | Keiya Ozawa | Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions |
| CA2426283C (en) | 2002-04-29 | 2006-06-27 | The Trustees Of The University Of Pennsylvania | Method for direct rescue and amplification of integrated viruses from cellular dna of tissues |
| CA2482512C (en) | 2002-04-30 | 2011-09-20 | Oncolytics Biotech Inc. | Improved viral purification methods |
| WO2004027019A2 (en) | 2002-05-01 | 2004-04-01 | University Of Florida Research Foundation, Inc. | Improved raav expression systems for genetic modification of specific capsid proteins |
| WO2003093441A2 (en) | 2002-05-03 | 2003-11-13 | Duke University | A method of regulating gene expression |
| DE60335672D1 (de) | 2002-05-14 | 2011-02-17 | Merck Sharp & Dohme | Verfahren zur reinigung von adenovirus |
| US7419817B2 (en) | 2002-05-17 | 2008-09-02 | The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. | Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US20050255086A1 (en) | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
| US20040241854A1 (en) | 2002-08-05 | 2004-12-02 | Davidson Beverly L. | siRNA-mediated gene silencing |
| WO2004020605A2 (en) | 2002-08-29 | 2004-03-11 | The Board Of Trustees Of The Leland Stanford Junior University | Circular nucleic acid vectors, and methods for making and using the same |
| AU2003273336A1 (en) | 2002-09-18 | 2004-04-08 | Isis Pharmaceuticals, Inc. | Efficient reduction of target rna's by single- and double-stranded oligomeric compounds |
| US7892793B2 (en) | 2002-11-04 | 2011-02-22 | University Of Massachusetts | Allele-specific RNA interference |
| US20060041022A1 (en) | 2002-11-06 | 2006-02-23 | Pasinetti Giulio M | Treatment of amyotrophic lateral sclerosis with nimesulide |
| EP1418185A1 (en) | 2002-11-11 | 2004-05-12 | Aventis Pharma Deutschland GmbH | Use of EDG2 receptor in an animal model of heart failure |
| US7169612B2 (en) | 2002-11-11 | 2007-01-30 | Sanofi-Aventis Deutschland Gmbh | Use of EDG2 receptor in an animal model of heart failure |
| EP2305812A3 (en) | 2002-11-14 | 2012-06-06 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| WO2006006948A2 (en) | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
| US7618948B2 (en) | 2002-11-26 | 2009-11-17 | Medtronic, Inc. | Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA |
| JP2006523103A (ja) | 2003-02-21 | 2006-10-12 | ザ ペン ステイト リサーチ ファウンデーション | Ｒｎａ干渉組成物および方法 |
| WO2004075861A2 (en) | 2003-02-26 | 2004-09-10 | Children's Hospital, Inc. | Recombinant adeno-associated virus production |
| WO2004083441A2 (en) | 2003-03-19 | 2004-09-30 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Random peptide library displayed on aav vectors |
| WO2004108922A2 (en) | 2003-04-25 | 2004-12-16 | The Trustees Of The University Of Pennsylvania | Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver |
| WO2005017101A2 (en) | 2003-05-19 | 2005-02-24 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH | Avian adenoassociated virus (aaav) and uses thereof |
| DE602004030327D1 (de) | 2003-05-21 | 2011-01-13 | Genzyme Corp | Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind |
| DK1633767T3 (en) | 2003-06-02 | 2019-03-25 | Univ Massachusetts | METHODS AND COMPOSITIONS FOR MANAGING THE EFFECT OF RNA SILENCING |
| US7750144B2 (en) | 2003-06-02 | 2010-07-06 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of RNA silencing |
| ES2357116T5 (es) | 2003-06-02 | 2021-09-03 | Univ Massachusetts | Métodos y composiciones para mejorar la eficacia y especificad de FNAi |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| JP4888876B2 (ja) | 2003-06-13 | 2012-02-29 | 田平 武 | アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター |
| SI3235827T1 (sl) | 2003-06-19 | 2021-05-31 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen |
| US7291498B2 (en) | 2003-06-20 | 2007-11-06 | The Trustees Of The University Of Pennsylvania | Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses |
| WO2005001103A2 (en) | 2003-06-20 | 2005-01-06 | The Trustees Of The University Of Pennsylvania | Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| WO2005007875A2 (en) | 2003-07-18 | 2005-01-27 | University Of Massachusetts | Enhanced promoters for synthesis of small hairpin rna |
| ES2391975T3 (es) | 2003-07-25 | 2012-12-03 | Genvec, Inc. | Vacunas a base de vector adenovírico |
| US7683036B2 (en) | 2003-07-31 | 2010-03-23 | Regulus Therapeutics Inc. | Oligomeric compounds and compositions for use in modulation of small non-coding RNAs |
| WO2005019828A1 (en) | 2003-08-20 | 2005-03-03 | Amorfix Life Sciences Ltd. | Epitope protection assay and method for detecting protein conformations |
| WO2005027980A1 (en) | 2003-09-12 | 2005-03-31 | University Of Massachusetts | Rna interference for the treatment of gain-of-function disorders |
| US20050064489A1 (en) | 2003-09-24 | 2005-03-24 | Zhang Fang Liang | Engineered U6 and H1 promoters |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| CN1926551B (zh) | 2003-10-27 | 2010-06-16 | 罗斯塔生化科技有限责任公司 | 用于基因沉默的siRNA的设计方法 |
| WO2005062937A2 (en) | 2003-12-22 | 2005-07-14 | University Of Massachusetts | Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna |
| US20050182420A1 (en) | 2004-02-13 | 2005-08-18 | Schulte Gregory T. | Low profile apparatus for securing a therapy delivery device within a burr hole |
| AU2005222084B2 (en) | 2004-03-05 | 2010-04-22 | Benitec, Inc. | Multiple promoter expression cassettes for simultaneous delivery of RNAI agents |
| US7498316B2 (en) | 2004-04-06 | 2009-03-03 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using RNA interference |
| ATE497785T1 (de) | 2004-04-28 | 2011-02-15 | Univ Pennsylvania | Sequenzielle freisetzung immunogener moleküle über adenoviren und adeno-assoziierte viren vermittelte abgaben |
| GB0412072D0 (en) * | 2004-05-28 | 2004-06-30 | Syngenta Participations Ag | Chemical compounds |
| EP1751275B1 (en) | 2004-06-01 | 2017-08-16 | Avigen, Inc. | Compositions and methods to prevent aav vector aggregation |
| PT1807009E (pt) | 2004-10-05 | 2015-02-25 | Univ California | Cânula escalonada |
| US7901921B2 (en) | 2004-10-22 | 2011-03-08 | Oncolytics Biotech Inc. | Viral purification methods |
| WO2006055727A2 (en) | 2004-11-18 | 2006-05-26 | The Board Of Trustees Of The University Of Illinois | MULTICISTRONIC siRNA CONSTRUCTS TO INHIBIT TUMORS |
| CN1286981C (zh) | 2004-11-30 | 2006-11-29 | 华中科技大学同济医学院附属同济医院 | 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法 |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| US20060229268A1 (en) | 2004-12-16 | 2006-10-12 | Alsgen, Llc | Small interference RNA (siRNA) molecules for modulating superoxide dismutase (SOD) |
| JP4292416B2 (ja) | 2005-01-12 | 2009-07-08 | 住友電気工業株式会社 | 超電導ケーブルの端末構造 |
| CA2596711C (en) | 2005-02-03 | 2015-12-15 | Benitec, Inc. | Rnai expression constructs |
| US8614101B2 (en) | 2008-05-20 | 2013-12-24 | Rapid Pathogen Screening, Inc. | In situ lysis of cells in lateral flow immunoassays |
| US7625570B1 (en) | 2005-03-10 | 2009-12-01 | The Regents Of The University Of California | Methods for purifying adeno-associated virus |
| WO2006102072A2 (en) | 2005-03-23 | 2006-09-28 | The Trustees Of The University Of Pennsylvania | Use of a pa131 polypeptide in treatment of atherosclerosis |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| EP1879624B1 (en) | 2005-05-02 | 2011-09-21 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| EP2239328A3 (en) | 2005-08-18 | 2011-01-05 | Alnylam Pharmaceuticals Inc. | Methods and compositions for treating neurological disease |
| AU2006283189B2 (en) | 2005-08-23 | 2013-01-31 | The Regents Of The University Of California | Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery |
| EP1857552A1 (en) | 2006-05-20 | 2007-11-21 | Cargill Incorporated | Thermostable xylose isomerase enzyme |
| EP2311967B1 (en) | 2005-10-20 | 2017-09-20 | UniQure IP B.V. | Improved AAV vectors produced in insect cells |
| US7794692B2 (en) | 2005-12-02 | 2010-09-14 | Amorfix Life Sciences Ltd. | Methods and compositions for detecting amyotrophic lateral sclerosis |
| US7887803B2 (en) | 2005-12-02 | 2011-02-15 | Amorfix Life Sciences | Methods and compositions to treat misfolded-SOD1 mediated diseases |
| WO2007089607A2 (en) | 2006-01-26 | 2007-08-09 | University Of Massachusetts | Rna silencing agents for use in therapy and nanotransporters for efficient delivery of same |
| US7867484B2 (en) | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| WO2008016391A2 (en) | 2006-01-31 | 2008-02-07 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
| JP5823663B2 (ja) | 2006-03-03 | 2015-11-25 | プロミス ニューロサイエンシズ インコーポレイテッド | ミスフォールドｓｏｄ１媒介疾患を処置および検出するための方法および組成物 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| CA2648132C (en) | 2006-04-03 | 2019-05-28 | Santaris Pharma A/S | Pharmaceutical composition comprising anti-mirna antisense oligonucleotides |
| EP2018421B1 (en) | 2006-04-28 | 2012-12-19 | The Trustees of the University of Pennsylvania | Scalable production method for aav |
| CN101437538A (zh) | 2006-04-28 | 2009-05-20 | 宾夕法尼亚州立大学托管会 | 修饰的腺病毒六邻体蛋白和其应用 |
| WO2008027084A2 (en) | 2006-04-28 | 2008-03-06 | The Trustees Of The University Of Pennsylvania | Modified aav vectors having reduced capsid immunogenicity and use thereof |
| US20080003565A1 (en) | 2006-05-02 | 2008-01-03 | Government Of The Us, As Represented By The Secretary, Department Of Health And Human Services | Viral nucleic acid microarray and method of use |
| EP2023940B1 (en) | 2006-05-05 | 2011-06-22 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression of sglt2 |
| ES2465996T3 (es) | 2006-05-25 | 2014-06-09 | Sangamo Biosciences, Inc. | Métodos y composiciones para la inactivación genética |
| KR101589259B1 (ko) | 2006-06-21 | 2016-02-01 | 유니큐어 아이피 비.브이. | 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터 |
| BRPI0714495B8 (pt) | 2006-07-21 | 2021-05-25 | California Inst Of Techn | lentivírus deficiente para replicação recombinante pseudotipado |
| ES2385679T3 (es) | 2006-08-24 | 2012-07-30 | Virovek, Inc. | Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos |
| FR2905806B1 (fr) | 2006-09-13 | 2008-12-26 | Valeo Equip Electr Moteur | Arbre de rotor a griffes, rotor a griffes equipe d'un tel arbre et machine electrique tournante equipee d'un tel rotor |
| PL2066791T3 (pl) | 2006-10-03 | 2013-02-28 | Genzyme Corp | Terapia Genowa do leczenia stwardnienia zanikowego bocznego i innych zaburzeń rdzenia kręgowego |
| US7794443B2 (en) | 2006-10-24 | 2010-09-14 | Medtronic, Inc. | System and method for intraparenchymal drug infusion |
| CA2693178C (en) | 2006-11-29 | 2018-12-04 | Nationwide Children's Hospital, Inc. | Myostatin inhibition for enhancing muscle and/or improving muscle function |
| US8227592B2 (en) | 2006-11-29 | 2012-07-24 | University Of Iowa Research Foundation | Alternative export pathways for vector expressed RNA interference |
| US8183219B2 (en) | 2007-01-03 | 2012-05-22 | Medtronic, Inc. | Therapeuting compositions comprising an RNAi agent and a neurotrophic factor and methods of use thereof |
| US8173614B2 (en) | 2007-01-03 | 2012-05-08 | Medtronic, Inc. | Therapeuting compositions comprising an RNAi agent and a neurotrophic factor and methods of use thereof |
| US8071752B2 (en) | 2007-01-29 | 2011-12-06 | City Of Hope | Multi-targeting short interfering RNAs |
| SG178744A1 (en) | 2007-02-02 | 2012-03-29 | Biogen Idec Inc | Use of semaphorin 6a for promoting myelination and oligodendrocyte differentiation |
| DK2191001T3 (en) | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| US9611302B2 (en) | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of use |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| WO2008128251A1 (en) | 2007-04-17 | 2008-10-23 | The Children's Hospital Of Philadelphia | Humanized viral vectors and methods of use thereof |
| US8258286B2 (en) | 2007-04-26 | 2012-09-04 | University Of Iowa Research Foundation | Reduction of off-target RNA interference toxicity |
| EP2152874A2 (en) | 2007-04-26 | 2010-02-17 | University of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| EP2012122A1 (en) | 2007-07-06 | 2009-01-07 | Medigene AG | Mutated parvovirus structural proteins as vaccines |
| US20100203083A1 (en) | 2007-05-31 | 2010-08-12 | Medigene Ag | Mutated structural protein of a parvovirus |
| US8841437B2 (en) | 2008-06-20 | 2014-09-23 | The Board Of Trustees Of The Leland Stanford Junior University | Precursor miRNA loop-modulated target regulation |
| ATE535607T1 (de) | 2007-06-29 | 2011-12-15 | Hoffmann La Roche | Promotor |
| CN102149826A (zh) | 2007-06-29 | 2011-08-10 | 波士顿生物医药公司 | 通过微小rna装置进行细菌介导的基因调节 |
| WO2009012176A2 (en) | 2007-07-14 | 2009-01-22 | The University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| SI3093345T1 (sl) | 2007-07-26 | 2019-08-30 | Uniqure Ip B.V. | Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami |
| JP2010537640A (ja) | 2007-08-27 | 2010-12-09 | ボストン バイオメディカル， インコーポレイテッド | マイクロｒｎａ模倣剤または阻害剤としての非対称性ｒｎａ二重鎖の組成物 |
| WO2009030025A1 (en) | 2007-09-04 | 2009-03-12 | Her Majesty The Queen In Right Of Canada As Represented By The Minister Of Health | Porcine adeno-associated viruses |
| JP2010538675A (ja) | 2007-09-19 | 2010-12-16 | アムステルダム モレキュラー セラピューティクス ビー．ブイ． | タンパク質産生の改善のためのａａｖ複製機構の使用 |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| EP2186283A4 (en) | 2007-10-18 | 2011-03-09 | Lg Electronics Inc | METHOD AND SYSTEM FOR TRANSMITTING AND RECEIVING SIGNALS |
| EP2463362B1 (en) | 2007-11-28 | 2017-11-08 | The Trustees Of The University Of Pennsylvania | Simian subfamily c adenovirus SAdv-31 and uses thereof |
| MX2010005858A (es) | 2007-11-28 | 2010-06-11 | Univ Pennsylvania | Adenovirus e simianos sadv-39, sadv-25.2, sadv-26, sadv-30, sadv-37 y sadv-38. |
| CA2706257C (en) | 2007-11-28 | 2017-06-06 | The Trustees Of The University Of Pennsylvania | Simian subfamily b adenovirus sadv-28, -27, -29, -32, -33 and -35 and uses thereof |
| US8480626B2 (en) | 2007-11-30 | 2013-07-09 | Medtronic, Inc. | Infusion catheter assembly with reduced backflow |
| ES2414804T3 (es) | 2008-01-18 | 2013-07-22 | Genentech, Inc. | Métodos y composiciones que se dirigen a la poliubiquitina |
| DK2242840T3 (da) | 2008-01-29 | 2019-10-21 | Applied Genetic Tech Corporation | Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension |
| US10131904B2 (en) * | 2008-02-11 | 2018-11-20 | Rxi Pharmaceuticals Corporation | Modified RNAi polynucleotides and uses thereof |
| EP3257937B1 (en) | 2008-02-19 | 2022-08-03 | uniQure IP B.V. | Optimisation of expression of parvoviral rep and cap proteins in insect cells |
| CA2716928C (en) | 2008-03-04 | 2018-04-10 | The Trustees Of The University Of Pennsylvania | Simian adenoviruses sadv-36,-42.1, -42.2, and -44 and uses thereof |
| US8632764B2 (en) | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| WO2009134681A2 (en) | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
| EP2910637A1 (en) | 2008-05-20 | 2015-08-26 | University of Florida Research Foundation, Inc. | Vectors for delivery of light-sensitive proteins and methods of use |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| WO2009151620A2 (en) | 2008-06-13 | 2009-12-17 | Cornell Research Foundation, Inc. Wmc | Pain treatment using erk2 inhibitors |
| WO2009154452A1 (en) | 2008-06-17 | 2009-12-23 | Amsterdam Molecular Therapeutics B.V. | Parvoviral capsid with incorporated Gly-Ala repeat region |
| US8945885B2 (en) | 2008-07-03 | 2015-02-03 | The Board Of Trustees Of The Leland Stanford Junior University | Minicircle DNA vector preparations and methods of making and using the same |
| JP5328244B2 (ja) | 2008-07-07 | 2013-10-30 | 株式会社ニュージェン・ファーマ | 筋萎縮性側索硬化症治療剤 |
| EP2318528A1 (en) | 2008-07-24 | 2011-05-11 | Rxi Pharmaceuticals Corporation | Rnai constructs and uses therof |
| US8212019B2 (en) | 2008-07-30 | 2012-07-03 | University Of Massachusetts | Nucleic acid silencing sequences |
| CA2746527A1 (en) | 2008-09-22 | 2010-03-25 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| AU2009297243B2 (en) | 2008-09-29 | 2015-10-29 | Proyecto De Biomedicina Cima S.L. | Porphobilinogen deaminase gene therapy |
| WO2010048446A2 (en) | 2008-10-22 | 2010-04-29 | Genentech, Inc. | Modulation of axon degeneration |
| US8940290B2 (en) | 2008-10-31 | 2015-01-27 | The Trustees Of The University Of Pennsylvania | Simian adenoviruses SAdV-43, -45, -46, -47, -48, -49, and -50 and uses thereof |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| PL2403867T3 (pl) | 2009-03-04 | 2019-12-31 | Deutsches Krebsforschungszentrum | Białko aktywujące składanie (aap) i jego zastosowanie do wytwarzania cząstek parwowirusa zasadniczo składających się z vp3 |
| US20120093775A1 (en) | 2009-03-27 | 2012-04-19 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
| ES2724122T3 (es) | 2009-04-30 | 2019-09-06 | Univ Pennsylvania | Composiciones para dirigir células de las vías respiratorias de conducción que comprenden construcciones de virus adenoasociado |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US8476418B2 (en) | 2009-05-28 | 2013-07-02 | Deutsches Krebsforschungszentrum | Modified AAV capsid polypeptides |
| CN102575232B (zh) | 2009-05-29 | 2015-07-22 | 宾夕法尼亚大学托管会 | 猿腺病毒41及其应用 |
| HUE028341T2 (en) | 2009-06-16 | 2016-12-28 | Genzyme Corp | Improved methods for purification of recombinant aav vectors |
| JP2014501097A (ja) | 2009-07-06 | 2014-01-20 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | 生物由来物質の産生を高めるための組成物及び方法 |
| EP3659435A1 (en) | 2009-07-15 | 2020-06-03 | Calimmune, Inc. | Dual vector for inhibition of human immunodeficiency virus |
| EP2292781A1 (en) | 2009-08-17 | 2011-03-09 | Genethon | Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions |
| WO2011038187A1 (en) | 2009-09-25 | 2011-03-31 | The Trustees Of The University Of Pennsylvania | Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom |
| WO2011054976A2 (en) | 2009-11-09 | 2011-05-12 | Genepod Therapeutics Ab | Novel viral vector construct for neuron specific optimized continuous dopa synthesis in vivo |
| RU2577971C2 (ru) | 2009-11-19 | 2016-03-20 | Нэшнл Юниверсити Корпорейшн Окаяма Юниверсити | Система для увеличения экспрессии генов и вектор, содержащий указанную систему |
| WO2011069529A1 (en) | 2009-12-09 | 2011-06-16 | Curevac Gmbh | Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids |
| ES2683695T3 (es) | 2010-01-12 | 2018-09-27 | The University Of North Carolina At Chapel Hill | Repeticiones terminales invertidas restrictivas para vectores virales |
| CA2788682C (en) | 2010-02-05 | 2019-03-05 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| US9163261B2 (en) | 2010-02-22 | 2015-10-20 | Koteswara Rao KOLLIPARA | Adeno-associated virus 2/8—micro RNA-101 therapy for liver cancer |
| WO2011112089A2 (en) | 2010-03-11 | 2011-09-15 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Mutated rep encoding sequences for use in aav production |
| WO2011117258A2 (en) | 2010-03-22 | 2011-09-29 | Association Institut De Myologie | Methods of increasing efficiency of vector penetration of target tissue |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| WO2011122950A1 (en) | 2010-04-01 | 2011-10-06 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Monomeric duplex aav vectors |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| EP2561075B1 (en) | 2010-04-23 | 2018-06-27 | University of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US9839696B2 (en) | 2010-04-30 | 2017-12-12 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2611437B1 (en) | 2010-09-02 | 2017-03-29 | Kyoto University | Pharmaceutical composition for prevention and treatment of amyotrophic lateral sclerosis |
| US8808684B2 (en) | 2010-09-10 | 2014-08-19 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction |
| US9102943B2 (en) | 2010-10-05 | 2015-08-11 | Takara Bio Inc. | Method for producing virus vector |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| WO2012064920A1 (en) | 2010-11-11 | 2012-05-18 | University Of Miami | Methods, compositions, cells, and kits for treating ischemic injury |
| WO2012071318A2 (en) | 2010-11-23 | 2012-05-31 | The Trustees Of The University Of Pennsylvania | Subfamily e simian adenoviruses a1321, a1325, a1295, a1309, a1316 and a1322 and uses thereof |
| ES2739804T3 (es) | 2011-02-12 | 2020-02-04 | Univ Iowa Res Found | Compuestos terapéuticos |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| GB201103062D0 (en) | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| EP2500434A1 (en) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| CN103476399A (zh) | 2011-04-18 | 2013-12-25 | 独立行政法人国立精神·神经医疗研究中心 | 药剂递送粒子及其制造方法 |
| EP2699688A1 (en) | 2011-04-20 | 2014-02-26 | The Trustees Of The University Of Pennsylvania | Regimens and compositions for aav-mediated passive immunization of airborne pathogens |
| EP3318635A1 (en) | 2011-04-21 | 2018-05-09 | University of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| WO2012149646A1 (en) | 2011-05-05 | 2012-11-08 | Sunnybrook Research Institute | Mirna inhibitors and their uses |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| US9598468B2 (en) | 2011-05-18 | 2017-03-21 | University Of Florida Research Foundation, Incorporated | Polypeptides and vectors for targeting HER2/neu expressing cells and uses thereof |
| CA2831969A1 (en) | 2011-06-06 | 2012-12-30 | Biocartis S.A. | Selective lysis of cells by ionic surfactants |
| US20130019580A1 (en) | 2011-07-20 | 2013-01-24 | Anderson Noel W | Bidirectional harvesting system |
| AU2012286994B2 (en) | 2011-07-25 | 2017-08-10 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of DUX4 |
| EP2737071B1 (en) | 2011-07-27 | 2017-03-15 | Genethon | Improved baculovirus expression systems |
| US8852911B2 (en) | 2011-08-04 | 2014-10-07 | The Regents Of The University Of California | Method of producing dicer |
| PT2744895E (pt) | 2011-09-08 | 2016-02-08 | Uniqure Ip Bv | Remoção de vírus contaminantes de preparações de aav |
| WO2013036889A1 (en) | 2011-09-09 | 2013-03-14 | University Of Washington | Retrograde transport peptide and use of same for delivery to central nervous system |
| US9441206B2 (en) | 2011-10-28 | 2016-09-13 | The University Of North Carolina At Chapel Hill | Cell line for production of adeno-associated virus |
| JP6348064B2 (ja) | 2011-11-22 | 2018-06-27 | ザ チルドレンズ ホスピタル オブ フィラデルフィア | 効率の高いトランスジーン送達のためのウイルスベクター |
| WO2013078199A2 (en) | 2011-11-23 | 2013-05-30 | Children's Medical Center Corporation | Methods for enhanced in vivo delivery of synthetic, modified rnas |
| US9611305B2 (en) | 2012-01-06 | 2017-04-04 | Mayo Foundation For Medical Education And Research | Treating cardiovascular or renal diseases |
| CN108310396B (zh) | 2012-02-14 | 2022-07-15 | 加利福尼亚大学董事会 | 用于心血管疾病和其它病况的旁分泌基因的全身性递送和受调节的表达 |
| AU2013221212B2 (en) | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| AU2013249202B2 (en) | 2012-04-18 | 2018-08-09 | The Children's Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using AAV capsid variants |
| US20140162319A2 (en) | 2012-05-02 | 2014-06-12 | Sangeetha Hareendran | Nucleotide sequences, methods, kit and a recombinant cell thereof |
| US9163259B2 (en) | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| WO2013170078A1 (en) | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| TWI775096B (zh) | 2012-05-15 | 2022-08-21 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| WO2013173702A2 (en) | 2012-05-18 | 2013-11-21 | The Trustees Of The University Of Pennsylvania | Subfamily e simian adenoviruses a1302, a1320, a1331 and a1337 and uses thereof |
| WO2013177367A2 (en) | 2012-05-23 | 2013-11-28 | The Johns Hopkins University | Compounds and methods of use thereof for treating neurodegenerative disorders |
| BR112015000161A2 (pt) | 2012-07-06 | 2017-06-27 | Univ Iowa Res Found | composições de vetor de vírus adeno-associado modificado |
| US9422576B2 (en) | 2012-07-06 | 2016-08-23 | Takara Bio Inc. | Cell capable of producing adeno-associated virus vector |
| CA2879514C (en) | 2012-07-17 | 2020-04-14 | Universite De Geneve | Nucleic acids for down-regulation of gene expression |
| AU2013296425B2 (en) | 2012-08-01 | 2018-06-07 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| AU2013315007A1 (en) | 2012-09-17 | 2015-04-09 | The Research Institute At Nationwide Children's Hospital | Compositions and methods for treating amyotrophic lateral sclerosis |
| US9636370B2 (en) | 2012-09-28 | 2017-05-02 | The University Of North Carolina At Chapel Hill | AAV vectors targeted to oligodendrocytes |
| US20140243783A1 (en) | 2012-10-06 | 2014-08-28 | Raghu Raghavan | Method of backflow reduction during material delivery through a needle into tissue |
| AU2013204200B2 (en) | 2012-10-11 | 2016-10-20 | Brandeis University | Treatment of amyotrophic lateral sclerosis |
| WO2014071042A1 (en) | 2012-10-31 | 2014-05-08 | The Trustees Of Columbia University In The City Of New York | Methods for identifying candidates for the treatment of neurodegenerative diseases |
| WO2014071219A1 (en) | 2012-11-01 | 2014-05-08 | Factor Bioscience Inc. | Methods and products for expressing proteins in cells |
| DK2931897T3 (en) | 2012-12-12 | 2018-02-05 | Broad Inst Inc | CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS |
| EP2940131B1 (en) | 2012-12-25 | 2019-02-20 | Takara Bio Inc. | Aav variant |
| CN105143450A (zh) | 2013-01-08 | 2015-12-09 | 贝尼泰克生物制药有限公司 | 年龄相关性黄斑变性治疗 |
| CA2897444A1 (en) | 2013-01-08 | 2014-07-17 | Genzyme Corporation | Use of inos inhibitors to increase viral yield in culture |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| EP2970946A4 (en) | 2013-03-13 | 2016-09-07 | Philadelphia Children Hospital | ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF |
| MX384795B (es) | 2013-03-15 | 2025-03-14 | Childrens Hospital Philadelphia | Vectores que comprenden secuencias de polinucleotidos rellenadoras/de relleno y metodos de uso. |
| WO2014144844A1 (en) | 2013-03-15 | 2014-09-18 | The Board Of Trustees Of The Leland Stanford Junior University | tRNA DERIVED SMALL RNAs (tsRNAs) INVOLVED IN CELL VIABILITY |
| US9447433B2 (en) | 2013-03-15 | 2016-09-20 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats |
| AU2014251099B2 (en) | 2013-04-08 | 2019-01-17 | The University Of Kansas | Chimeric adeno-associated virus/ bocavirus parvovirus vector |
| EP2792742A1 (en) | 2013-04-17 | 2014-10-22 | Universitätsklinikum Hamburg-Eppendorf (UKE) | Gene-therapy vectors for treating cardiomyopathy |
| CA2909733C (en) | 2013-04-18 | 2023-10-17 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| KR102413498B1 (ko) | 2013-04-20 | 2022-06-24 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달 |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| WO2014186746A1 (en) | 2013-05-16 | 2014-11-20 | University Of Florida Research Foundation, Inc. | HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| WO2014201308A1 (en) | 2013-06-12 | 2014-12-18 | Washington University | Endothelial-targeted adenoviral vectors, methods and uses therefor |
| EP3008191A2 (en) | 2013-06-13 | 2016-04-20 | Shire Human Genetic Therapies, Inc. | Messenger rna based viral production |
| KR20240090694A (ko) | 2013-07-22 | 2024-06-21 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| EP3024497B1 (en) | 2013-07-26 | 2021-01-13 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| ITTO20130669A1 (it) | 2013-08-05 | 2015-02-06 | Consiglio Nazionale Ricerche | Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari |
| JP2016527900A (ja) | 2013-08-14 | 2016-09-15 | キアゲン マンスフィールド インコーポレイテッド | ｃＭＥＴ核酸のマルチモード解析のための組成物および方法 |
| LT3702466T (lt) | 2013-08-27 | 2023-02-27 | Research Institute At Nationwide Children's Hospital | Produktai ir būdai, skirti šoninės amiotrofinės sklerozės gydymui |
| EP3039129B1 (en) | 2013-08-30 | 2018-06-27 | Amgen Inc. | High titer recombinant aav vector production in adherent and suspension cells |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| US20160354489A1 (en) | 2013-09-26 | 2016-12-08 | Universitat Autònome de Barcelona | Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease |
| WO2015048534A1 (en) | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| HUE052676T2 (hu) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| CN120174012A (zh) | 2013-10-24 | 2025-06-20 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| WO2015069647A1 (en) | 2013-11-05 | 2015-05-14 | The Research Institute At Nationwide Children's Hospital | COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS |
| WO2015108610A1 (en) | 2013-11-08 | 2015-07-23 | The Board Of Trustees Of The University Of Arkansas | Adeno-associated virus "x" oncogene |
| WO2015081101A1 (en) | 2013-11-26 | 2015-06-04 | The United States Of America, As Represented By The Secretary Department Of Health And Human Services | Adeno-associated virus vectors for treatment of glycogen storage disease |
| CN105765066B (zh) | 2013-11-29 | 2018-12-14 | 宝生物工程株式会社 | 用于定量腺伴随病毒的方法 |
| US20170002350A1 (en) | 2013-12-03 | 2017-01-05 | Agency For Science, Technology And Research | Tailed mirtron effectors for rnai-mediated gene silencing |
| EP4674963A3 (en) | 2013-12-09 | 2026-04-29 | Baylor College of Medicine | Hippo and dystrophin complex signaling in cardiomyocyte renewal |
| MX2016007325A (es) | 2013-12-12 | 2017-07-19 | Broad Inst Inc | Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. |
| GB201322798D0 (en) | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| WO2015106273A2 (en) | 2014-01-13 | 2015-07-16 | Trustees Of Boston University | Methods and assays relating to huntingtons disease and parkinson's disease |
| GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| WO2015124546A1 (en) | 2014-02-19 | 2015-08-27 | Fundación Centro Nacional De Investigaciones Cardiovasculares Carlos Iii- Cnic | Aav vectors for the treatment of ischemic and non-ischemic heart disease |
| WO2015126972A1 (en) | 2014-02-21 | 2015-08-27 | University Of Florida Research Foundation, Inc. | Methods and compositions for gene delivery to on bipolar cells |
| ES2768763T3 (es) | 2014-03-04 | 2020-06-23 | Univ Florida | Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR |
| WO2015137802A1 (en) | 2014-03-10 | 2015-09-17 | Uniqure Ip B.V. | Further improved aav vectors produced in insect cells |
| EP4410805A3 (en) | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| RS60707B1 (sr) | 2014-04-01 | 2020-09-30 | Biogen Ma Inc | Kompozicije za modulaciju ekspresije sod-1 |
| EP2933335A1 (en) | 2014-04-18 | 2015-10-21 | Genethon | A method of treating peripheral neuropathies and motor neuron diseases |
| ES2876409T3 (es) | 2014-04-25 | 2021-11-12 | Univ Pennsylvania | Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol |
| EP3134522B1 (en) | 2014-04-25 | 2021-10-06 | University of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| US9194250B1 (en) | 2014-05-07 | 2015-11-24 | General Electric Company | Embedded wireless sensors for turbomachine component defect monitoring |
| RU2691102C2 (ru) | 2014-05-08 | 2019-06-11 | Сангамо Байосайенсиз, Инк. | Способы и композиции для лечения болезни хантингтона |
| WO2015173436A1 (en) | 2014-05-16 | 2015-11-19 | Vrije Universiteit Brussel | Genetic correction of myotonic dystrophy type 1 |
| JP6663859B2 (ja) | 2014-05-20 | 2020-03-13 | ユニバーシティー オブ アイオワ リサーチ ファウンデーション | ハンチントン病の治療化合物 |
| EP3160980B1 (en) | 2014-05-28 | 2020-05-20 | The Regents of the University of California | HYBRID tRNA/pre-miRNA MOLECULES AND METHODS OF USE |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US11207424B2 (en) | 2014-06-13 | 2021-12-28 | Mayo Foundation For Medical Education And Research | Methods and materials for increasing viral vector infectivity |
| US10781459B2 (en) | 2014-06-20 | 2020-09-22 | University Of Florida Research Foundation, Incorporated | Methods of packaging multiple adeno-associated virus vectors |
| US10526583B2 (en) | 2014-07-02 | 2020-01-07 | University Of Florida Research Foundation, Incorporated | Compositions and methods for purifying recombinant adeno-associated virus |
| EP3168298B1 (en) | 2014-07-10 | 2019-04-17 | Takara Bio Inc. | Production method for non-enveloped virus particles |
| WO2016016449A1 (en) | 2014-07-31 | 2016-02-04 | Association Institut De Myologie | Treatment of amyotrophic lateral sclerosis |
| US10392622B2 (en) | 2014-08-01 | 2019-08-27 | The Trustees Of The University Of Pennsylvania | Compositions and methods for self-regulated inducible gene expression |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| US10711270B2 (en) | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
| WO2016057975A2 (en) | 2014-10-10 | 2016-04-14 | Research Institute At Nationwide Children's Hospital | Guided injections for aav gene transfer to muscle |
| AU2015335923B2 (en) | 2014-10-21 | 2021-04-29 | University Of Massachusetts | Recombinant AAV variants and uses thereof |
| EP3215602B1 (en) | 2014-11-05 | 2019-12-25 | The Research Institute at Nationwide Children's Hospital | Methods and materials for producing recombinant viruses in eukaryotic microalgae |
| WO2016077607A1 (en) | 2014-11-13 | 2016-05-19 | The Regents Of The University Of California | Adjustable stepped cannula |
| CN119876138A (zh) | 2014-11-14 | 2025-04-25 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| WO2016081811A1 (en) | 2014-11-21 | 2016-05-26 | The University Of North Carolina At Chapel Hill | Aav vectors targeted to the central nervous system |
| WO2016081927A2 (en) | 2014-11-21 | 2016-05-26 | University Of Florida Research Foundation, Inc. | Genome-modified recombinant adeno-associated virus vectors |
| FI3224376T4 (fi) | 2014-11-28 | 2023-08-31 | Dna-epäpuhtaudet koostumuksessa, joka käsittää parvovirusvirionin | |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| AU2015370903B2 (en) | 2014-12-24 | 2021-06-17 | Uniqure Ip B.V. | RNAi induced huntingtin gene suppression |
| WO2016109649A1 (en) | 2014-12-30 | 2016-07-07 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| AU2016206624B2 (en) | 2015-01-14 | 2020-08-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for targeted gene transfer |
| EP3245221A4 (en) | 2015-01-16 | 2018-06-13 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
| CA2975447C (en) | 2015-01-30 | 2021-02-23 | The Regents Of The University Of California | Spinal subpial gene delivery system |
| WO2016126857A1 (en) | 2015-02-03 | 2016-08-11 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
| TN2017000353A1 (en) | 2015-02-10 | 2019-01-16 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| TN2017000354A1 (en) | 2015-02-10 | 2019-01-16 | Genzyme Corp | VARIANT RNAi |
| EP3262162A4 (en) | 2015-02-23 | 2018-08-08 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| JP2018510160A (ja) | 2015-03-20 | 2018-04-12 | ブルーバード バイオ， インコーポレイテッド | ベクター製剤 |
| JP6836999B2 (ja) | 2015-03-24 | 2021-03-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ | アデノ随伴ウイルス変異体及びその使用方法 |
| EP3277811B1 (en) | 2015-04-03 | 2020-12-23 | University of Massachusetts | Fully stabilized asymmetric sirna |
| SMT202000454T1 (it) | 2015-04-03 | 2020-11-10 | Univ Massachusetts | Cmposti oligonucleotidici per bersagliare l'mrn della huntingtina |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| CN115074366A (zh) | 2015-04-16 | 2022-09-20 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| CA3019315A1 (en) | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| US20160346359A1 (en) | 2015-05-01 | 2016-12-01 | Spark Therapeutics, Inc. | Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease |
| US10881548B2 (en) | 2015-05-07 | 2021-01-05 | Massachusetts Eye And Ear Infirmary | Methods of delivering an agent to the eye |
| CA2985347A1 (en) | 2015-05-11 | 2016-11-17 | Alcyone Lifesciences, Inc. | Drug delivery systems and methods |
| JP6805174B2 (ja) | 2015-05-12 | 2020-12-23 | アメリカ合衆国 | 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質 |
| US20170067028A1 (en) | 2015-05-15 | 2017-03-09 | Douglas J. Ballon | Radiolabeling of adeno associated virus |
| WO2016191418A1 (en) | 2015-05-26 | 2016-12-01 | Salk Institute For Biological Studies | Motor neuron-specific expression vectors |
| US11542500B2 (en) | 2015-05-29 | 2023-01-03 | The Trustees Of The University Of Pennsylvania | TRIM11 for degradation of protein aggregates |
| US11027024B2 (en) | 2015-05-29 | 2021-06-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
| WO2017004514A1 (en) | 2015-07-02 | 2017-01-05 | University Of Florida Research Foundation, Inc. | Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma |
| US20170007669A1 (en) | 2015-07-07 | 2017-01-12 | Mayo Foundation For Medical Education And Research | Peptide-mediated delivery of active agents across the blood-brain barrier |
| US10801040B2 (en) | 2015-07-07 | 2020-10-13 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject |
| US20170009304A1 (en) | 2015-07-07 | 2017-01-12 | Splicingcodes.Com | Method and kit for detecting fusion transcripts |
| WO2017015102A1 (en) | 2015-07-17 | 2017-01-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for achieving high levels of transduction in human liver cells |
| WO2017019876A1 (en) | 2015-07-28 | 2017-02-02 | University Of Massachusetts | Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector |
| CA2994160C (en) | 2015-07-30 | 2021-08-10 | Massachusetts Eye And Ear Infirmary | Ancestral virus sequences and uses thereof |
| WO2017024111A1 (en) | 2015-08-04 | 2017-02-09 | The University Of Chicago | Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6 |
| US10047377B2 (en) | 2015-09-22 | 2018-08-14 | Loyola University Of Chicago | Methods for modulating KLHL1 levels, methods for modulating current activity in T-type calcium channels, molecules therefor, and methods for identifying molecules therefor |
| BR112018003665A2 (en) | 2015-09-28 | 2018-09-25 | The University Of North Carolina At Chapel Hill | methods and compositions for antibody evasion viral vectors |
| US11635363B2 (en) | 2015-10-09 | 2023-04-25 | Genzyme Corporation | FLARE (flow cytometry attenuated reporter expression) technology for rapid bulk sorting |
| WO2017062983A1 (en) | 2015-10-09 | 2017-04-13 | The Children's Hospital Of Philadelphia | Compositions and methods for treating huntington's disease and related disorders |
| US10123969B2 (en) | 2015-10-15 | 2018-11-13 | Wisconsin Alumni Research Foundation | Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid |
| WO2017070476A2 (en) | 2015-10-22 | 2017-04-27 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav3 capsid library |
| WO2017070525A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
| CA3002980A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| US20180236105A1 (en) | 2015-10-23 | 2018-08-23 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| US20180230489A1 (en) | 2015-10-28 | 2018-08-16 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| EP3371217B1 (en) | 2015-11-08 | 2025-06-11 | F. Hoffmann-La Roche AG | Methods of screening for multispecific antibodies |
| US10633662B2 (en) | 2015-11-10 | 2020-04-28 | The Board Of Trustees Of The Leland Stanford Junior University | Methods and compositions for modulating AAV infection |
| AU2016362317B2 (en) | 2015-12-01 | 2023-03-16 | Spark Therapeutics, Inc. | Scalable methods for producing recombinant Adeno-Associated Viral (AAV) vector in serum-free suspension cell culture system suitable for clinical use |
| WO2017095753A1 (en) | 2015-12-01 | 2017-06-08 | Invivo Therapeutics Corporation | Compositions and methods for preparing an injectable medium for administration into the central nervous system |
| US10406244B2 (en) | 2015-12-02 | 2019-09-10 | The Board Of Trustees Of The Leland Stanford Junior University | AAV vectors with expanded packaging capacity |
| US10385320B2 (en) | 2015-12-02 | 2019-08-20 | The Board Of Trustees Of The Leland Stanford Junior University | Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
| CN108699135B (zh) | 2015-12-03 | 2022-10-04 | 吉尼松公司 | 用于改善病毒载体效率的组合物和方法 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| EP4659816A3 (en) | 2015-12-14 | 2025-12-31 | The University of North Carolina at Chapel Hill | MODIFIED CAPSID PROTEINS FOR IMPROVED PARVOVIRUS VECTOR DELIVERY |
| US11208630B2 (en) | 2015-12-24 | 2021-12-28 | University Of Florida Research Foundation, Incorporated | AAV production using suspension adapted cells |
| JP6924441B2 (ja) | 2016-01-15 | 2021-08-25 | 学校法人自治医科大学 | てんかん治療のためのアデノ随伴ウイルスビリオン |
| WO2017136536A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| PL3411484T3 (pl) | 2016-02-05 | 2024-02-19 | Emory University | Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego |
| WO2017139381A1 (en) | 2016-02-08 | 2017-08-17 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| US10179176B2 (en) | 2016-02-16 | 2019-01-15 | The Board Of Trustees Of The Leland Stanford Junior University | Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies |
| WO2017147477A1 (en) | 2016-02-26 | 2017-08-31 | University Of Florida Research Foundation, Inc. | Aav heparin mutants that display significantly improved eye and brain transduction |
| WO2017152149A1 (en) | 2016-03-03 | 2017-09-08 | University Of Massachusetts | Closed-ended linear duplex dna for non-viral gene transfer |
| CA3198936A1 (en) | 2016-03-07 | 2017-09-14 | University Of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
| WO2017165859A1 (en) | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Modified viral capsid proteins |
| US10899839B2 (en) | 2016-03-28 | 2021-01-26 | Tire Regents of the University of California | Anti-Ryk antibodies and methods of using the same |
| PL3436593T3 (pl) | 2016-03-28 | 2023-03-27 | Ultragenyx Pharmaceutical Inc. | Sposoby inaktywacji termicznej adenowirusów |
| MX2018011928A (es) | 2016-03-30 | 2019-03-28 | Spark Therapeutics Inc | Linea celular para produccion de proteina recombinante y/o vector viral. |
| BR112018070256A2 (pt) | 2016-03-31 | 2019-01-29 | Spark Therapeutics, Inc. | processo de fabricação de raav totalmente escalável à base de colunas |
| WO2017180854A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Novel aav8 mutant capsids and compositions containing same |
| WO2017181162A1 (en) | 2016-04-16 | 2017-10-19 | University Of Florida Research Foundation, Incorporated | Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus |
| KR20230079511A (ko) | 2016-04-21 | 2023-06-07 | 바이로베크 인코포레이티드 | 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물 |
| EP3235516B1 (en) | 2016-04-22 | 2019-06-26 | Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts Universitätsmedizin | Regulatable adeno-associated virus (aav) vector |
| EP3449250B1 (en) | 2016-04-28 | 2020-11-04 | Indiana University Research & Technology Corporation | Methods and compositions for resolving components of a virus preparation |
| EP3448875A4 (en) | 2016-04-29 | 2020-04-08 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| AU2017261249B2 (en) | 2016-05-03 | 2021-05-06 | Children's Medical Research Institute | Adeno-associated virus polynucleotides, polypeptides and virions |
| WO2017192750A1 (en) | 2016-05-04 | 2017-11-09 | Oregon Health & Science University | Recombinant adeno-associated viral vectors |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| CA3024449A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| JP2020518266A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 調節性ポリヌクレオチド |
| EP3635121A1 (en) | 2017-06-02 | 2020-04-15 | Institut National de la Sante et de la Recherche Medicale (INSERM) | Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖを送達するための組成物および方法 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| TW202015742A (zh) | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | 投遞腺相關病毒（ａａｖ）之組成物和方法 |
| WO2020010035A1 (en) | 2018-07-02 | 2020-01-09 | Voyager Therapeutics, Inc. | Cannula system |
| US20210254103A1 (en) | 2018-07-02 | 2021-08-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| WO2020223296A1 (en) | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| WO2023092002A2 (en) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis and disorders associatedwith the spinal cord |
-
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Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007109097A2 (en) * | 2006-03-16 | 2007-09-27 | Alnylam Pharmaceuticals, Inc. | RNAi MODULATION OF TGF-BETA AND THERAPEUTIC USES THEREOF |
| JP2013143917A (ja) * | 2012-01-13 | 2013-07-25 | Mie Univ | 線維症予防又は治療剤 |
Non-Patent Citations (3)
| Title |
|---|
| CHRIS TOWNE ET AL., MOLECULAR THERAPY, vol. 16, no. 6, JPN6019041063, 2008, pages 1018 - 1025, ISSN: 0004141221 * |
| TAKAYUKI KUBODERA ET AL., HUMAN GENE THERAPY, vol. 22, JPN6019041064, 2011, pages 27 - 34, ISSN: 0004141222 * |
| TIMOTHY M. MILLER ET AL., ANN NEUROL., vol. 57, no. 5, JPN6019041061, May 2005 (2005-05-01), pages 773 - 776, ISSN: 0004141220 * |
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