ATE428777T1 - Muskelspezifische expressionsvektoren - Google Patents
Muskelspezifische expressionsvektorenInfo
- Publication number
- ATE428777T1 ATE428777T1 AT02739449T AT02739449T ATE428777T1 AT E428777 T1 ATE428777 T1 AT E428777T1 AT 02739449 T AT02739449 T AT 02739449T AT 02739449 T AT02739449 T AT 02739449T AT E428777 T1 ATE428777 T1 AT E428777T1
- Authority
- AT
- Austria
- Prior art keywords
- muscle
- promoter
- elements
- expression vectors
- specific expression
- Prior art date
Links
- 239000013604 expression vector Substances 0.000 title 1
- 210000003205 muscle Anatomy 0.000 abstract 4
- 239000003623 enhancer Substances 0.000 abstract 3
- 102100036912 Desmin Human genes 0.000 abstract 2
- 108010044052 Desmin Proteins 0.000 abstract 2
- 108010059343 MM Form Creatine Kinase Proteins 0.000 abstract 2
- 102000013394 Troponin I Human genes 0.000 abstract 2
- 108010065729 Troponin I Proteins 0.000 abstract 2
- 210000005045 desmin Anatomy 0.000 abstract 2
- 102000007469 Actins Human genes 0.000 abstract 1
- 108010085238 Actins Proteins 0.000 abstract 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 abstract 1
- 210000000107 myocyte Anatomy 0.000 abstract 1
- 230000002085 persistent effect Effects 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Hematology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Obesity (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US29330401P | 2001-05-24 | 2001-05-24 | |
| PCT/US2002/016748 WO2002095006A2 (en) | 2001-05-24 | 2002-05-22 | Muscle-specific expression vectors |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE428777T1 true ATE428777T1 (de) | 2009-05-15 |
Family
ID=23128549
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT02739449T ATE428777T1 (de) | 2001-05-24 | 2002-05-22 | Muskelspezifische expressionsvektoren |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US20030100526A1 (de) |
| EP (2) | EP2017338A1 (de) |
| JP (1) | JP2004535801A (de) |
| AT (1) | ATE428777T1 (de) |
| CA (1) | CA2448120A1 (de) |
| DE (1) | DE60231975D1 (de) |
| WO (1) | WO2002095006A2 (de) |
Families Citing this family (67)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2406687A1 (en) * | 2001-11-09 | 2003-05-09 | Transgene S.A. | Chimeric promoters for controlling expression in muscle cells |
| US20080044393A1 (en) * | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
| US8557969B2 (en) | 2004-12-01 | 2013-10-15 | The Regents Of The University Of Michigan | Compositions and methods for regulating cardiac performance |
| WO2008054713A2 (en) * | 2006-10-30 | 2008-05-08 | Thomas Jefferson University | Tissue specific gene therapy treatment |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| EP2694114A1 (de) | 2011-04-06 | 2014-02-12 | Université Pierre et Marie Curie (Paris 6) | Skelettmuskelspezifischer verstärker |
| RS61055B1 (sr) * | 2012-06-19 | 2020-12-31 | Univ Florida | Kompozicije i postupci za lečenje bolesti |
| WO2014028762A1 (en) | 2012-08-15 | 2014-02-20 | University Of Virginia Patent Foundation | Compositions and methods for treating peripheral arterial disease |
| CA2894899A1 (en) | 2012-12-20 | 2014-06-26 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| RS58529B1 (sr) | 2013-03-14 | 2019-04-30 | Sarepta Therapeutics Inc | Sastavi koji preskaču egzon za tretiranje mišićne distrofije |
| NZ731587A (en) | 2013-03-14 | 2021-07-30 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| US20160220647A1 (en) * | 2013-09-09 | 2016-08-04 | University Of Washington Through Its Center For Commercialization | Proteins for targeting neuronal nitric oxide synthase to muscle sarcolemma and related methods of use |
| EP3097197B1 (de) * | 2014-01-21 | 2020-12-16 | Vrije Universiteit Brussel | Muskelspezifische regulatorische nukleinsäureelemente sowie verfahren und verwendung davon |
| CA2980819C (en) | 2015-03-05 | 2024-03-05 | Peter Und Traudl Engelhorn-Stiftung Zur Forderung Der Lebenswissenschaften | System for the presentation of peptides on the cell surface |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CN104894651B (zh) * | 2015-06-29 | 2017-04-12 | 天津诺禾医学检验所有限公司 | 微量起始dna的高通量测序文库构建方法及其所构建的高通量测序文库 |
| EA201890543A1 (ru) * | 2015-09-17 | 2018-08-31 | Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл | Способы и материалы для генной терапии galgt2 |
| CA3020754C (en) | 2016-04-14 | 2023-07-25 | Benitec Biopharma Limited | Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof |
| WO2017223128A1 (en) | 2016-06-21 | 2017-12-28 | The Curators Of The University Of Missouri | Modified dystrophin proteins |
| 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 |
| US12296026B2 (en) | 2018-02-05 | 2025-05-13 | Astellas Gene Therapies, Inc. | Transcription regulatory elements and uses thereof |
| EP3749771A1 (de) * | 2018-02-07 | 2020-12-16 | Genethon | Hybrid-regelelemente |
| CN112313331A (zh) | 2018-04-27 | 2021-02-02 | 沃雅戈治疗公司 | 用于测量aadc病毒载体效力的方法 |
| SG11202011296VA (en) | 2018-05-15 | 2020-12-30 | Voyager Therapeutics Inc | Compositions and methods for the treatment of parkinson's disease |
| 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 |
| US12060567B2 (en) | 2018-06-13 | 2024-08-13 | Voyager Therapeutics, Inc. | Engineered untranslated regions (UTR) for AAV production |
| US20210254103A1 (en) | 2018-07-02 | 2021-08-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| 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 |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| CA3116701A1 (en) | 2018-10-15 | 2020-04-23 | Voyager Therapeutics, Inc. | Expression vectors for large-scale production of raav in the baculovirus/sf9 system |
| EP3880255A1 (de) | 2018-11-16 | 2021-09-22 | Astellas Pharma Inc. | Verfahren zur behandlung von muskeldystrophie durch anvisierung des utrophin-gens |
| 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 |
| US20220098614A1 (en) | 2019-02-28 | 2022-03-31 | Benitec IP Holdings Inc. | Compositions and Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD) |
| US20220162640A1 (en) * | 2019-04-08 | 2022-05-26 | Genethon | Hybrid promoters for muscle expression |
| WO2020227515A1 (en) | 2019-05-07 | 2020-11-12 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| CA3135271A1 (en) | 2019-05-28 | 2020-12-03 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting dmpk gene |
| TW202112797A (zh) | 2019-08-16 | 2021-04-01 | 日商摩大力斯醫療公司 | 經由靶向lama1基因治療肌肉萎縮症的方法 |
| US20220333133A1 (en) | 2019-09-03 | 2022-10-20 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| WO2021202651A1 (en) | 2020-04-01 | 2021-10-07 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| MX2022012752A (es) | 2020-04-15 | 2023-01-16 | Voyager Therapeutics Inc | Compuestos de union a tau. |
| CN116096734A (zh) | 2020-05-13 | 2023-05-09 | 沃雅戈治疗公司 | Aav衣壳的向性的重定向 |
| WO2021230385A1 (en) | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| EP4211251A4 (de) * | 2020-09-11 | 2025-07-16 | Aldevron L L C | Muskelspezifischer hybridpromotor |
| US20220098615A1 (en) | 2020-09-30 | 2022-03-31 | NGGT, Inc. | Dual functional expression vectors and methods of use thereof |
| JP2023548746A (ja) | 2020-10-13 | 2023-11-21 | クリヤ セラピューティクス インコーポレイテッド | サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用 |
| AR124143A1 (es) | 2020-11-25 | 2023-02-15 | Astellas Pharma Inc | Método para tratar la distrofia muscular mediante el direccionamiento al gen dmpk |
| US20240092885A1 (en) | 2021-01-26 | 2024-03-21 | Kriya Therapeutics, Inc. | Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same |
| EP4036238A1 (de) * | 2021-01-27 | 2022-08-03 | The University Court of the University of Edinburgh | Funktionelles nukleinsäuremolekül und verfahren |
| IL304956A (en) | 2021-02-12 | 2023-10-01 | Merand Pharmaceuticals Inc | Agents, compositions, and methods for the treatment of hypoxia and ischemia-related disorders |
| US20240141383A1 (en) | 2021-03-04 | 2024-05-02 | Kriya Therapeutics, Inc. | Viral vector constructs incorporating dna for inhibiting toll like receptors and methods of using the same |
| CN117897492A (zh) * | 2021-08-04 | 2024-04-16 | 吉尼松公司 | 用于肌肉和cns中的基因表达的杂合启动子 |
| WO2023023055A1 (en) | 2021-08-16 | 2023-02-23 | Renagade Therapeutics Management Inc. | Compositions and methods for optimizing tropism of delivery systems for rna |
| TW202328067A (zh) | 2021-09-14 | 2023-07-16 | 美商雷納嘉德醫療管理公司 | 環狀脂質及其使用方法 |
| CN118317944A (zh) | 2021-09-14 | 2024-07-09 | 雷纳嘉德医疗管理公司 | 非环状脂质及其使用方法 |
| EP4405396A2 (de) | 2021-09-20 | 2024-07-31 | Voyager Therapeutics, Inc. | Zusammensetzungen und verfahren zur behandlung von her2-positivem krebs |
| JP2025503637A (ja) | 2022-01-09 | 2025-02-04 | クリヤ セラピューティクス， インコーポレイテッド | 治療用抗ｉｇｆ－１ｒ抗体をコードする核酸の送達のためのベクター構築物およびそれを使用する方法 |
| EP4493705A4 (de) * | 2022-03-16 | 2026-03-18 | Univ Rutgers | Kontrollierte muskelspezifische genverabreichung |
| CA3255619A1 (en) | 2022-04-07 | 2023-10-12 | Renagade Therapeutics Management Inc. | CYCLIC LIPIDS AND LIPID NANOPARTICLES (LPNs) FOR THE SUPPLY OF NUCLEIC ACIDS OR PEPTIDES INTENDED FOR USE IN VACCINATION AGAINST INFECTIOUS AGENTS |
| WO2023220695A2 (en) | 2022-05-13 | 2023-11-16 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| EP4543473A1 (de) | 2022-06-22 | 2025-04-30 | Voyager Therapeutics, Inc. | Tau-bindende verbindungen |
| JP2025526502A (ja) * | 2022-07-11 | 2025-08-13 | ヴァイタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための治療用サテライト細胞の製造 |
| CA3266790A1 (en) | 2022-09-15 | 2024-03-21 | Voyager Therapeutics, Inc. | TAU PROTEIN BINDING COMPOUNDS |
| WO2025014832A1 (en) | 2023-07-07 | 2025-01-16 | Kriya Therapeutics, Inc. | Periocular delivery of aav vectors for treating ophthalmic pathologies |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5266488A (en) * | 1988-04-06 | 1993-11-30 | The Regents Of The University Of California | Troponin T gene promoter and derivatives thereof |
| US5795872A (en) * | 1995-09-19 | 1998-08-18 | Pharmadigm, Inc. | DNA construct for immunization |
| US5858351A (en) * | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
| US5846528A (en) * | 1996-01-18 | 1998-12-08 | Avigen, Inc. | Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence |
| ES2219011T3 (es) * | 1998-04-24 | 2004-11-16 | University Of Florida | Vector viral adeno-asociado recombinante que codifica alfa-1 antitripsina para terapia genica. |
| ATE437944T1 (de) * | 2000-04-28 | 2009-08-15 | Asklepios Biopharmaceutical In | Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung |
| WO2001083703A2 (en) * | 2000-05-04 | 2001-11-08 | California Institute Of Technology | Checkpoint-activating oligonucleotides |
-
2002
- 2002-05-22 EP EP08103273A patent/EP2017338A1/de not_active Withdrawn
- 2002-05-22 JP JP2002592469A patent/JP2004535801A/ja active Pending
- 2002-05-22 EP EP02739449A patent/EP1390490B1/de not_active Expired - Lifetime
- 2002-05-22 AT AT02739449T patent/ATE428777T1/de not_active IP Right Cessation
- 2002-05-22 WO PCT/US2002/016748 patent/WO2002095006A2/en not_active Ceased
- 2002-05-22 DE DE60231975T patent/DE60231975D1/de not_active Expired - Lifetime
- 2002-05-22 CA CA002448120A patent/CA2448120A1/en not_active Abandoned
- 2002-05-24 US US10/156,604 patent/US20030100526A1/en not_active Abandoned
-
2007
- 2007-03-12 US US11/716,899 patent/US20110212529A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP1390490A4 (de) | 2005-05-11 |
| WO2002095006A2 (en) | 2002-11-28 |
| DE60231975D1 (de) | 2009-05-28 |
| JP2004535801A (ja) | 2004-12-02 |
| US20110212529A1 (en) | 2011-09-01 |
| EP1390490A2 (de) | 2004-02-25 |
| WO2002095006A3 (en) | 2003-03-13 |
| CA2448120A1 (en) | 2002-11-28 |
| EP1390490B1 (de) | 2009-04-15 |
| US20030100526A1 (en) | 2003-05-29 |
| EP2017338A1 (de) | 2009-01-21 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |