PL2191001T4 - Kompozycje wektora RAAV mające białka kapsydu zmodyfikowane tyrozyną i sposoby ich zastosowania - Google Patents

Kompozycje wektora RAAV mające białka kapsydu zmodyfikowane tyrozyną i sposoby ich zastosowania

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Publication number
PL2191001T4
PL2191001T4 PL08733161T PL08733161T PL2191001T4 PL 2191001 T4 PL2191001 T4 PL 2191001T4 PL 08733161 T PL08733161 T PL 08733161T PL 08733161 T PL08733161 T PL 08733161T PL 2191001 T4 PL2191001 T4 PL 2191001T4
Authority
PL
Poland
Prior art keywords
tyrosine
methods
capsid proteins
raav vector
modified capsid
Prior art date
Application number
PL08733161T
Other languages
English (en)
Other versions
PL2191001T3 (pl
Inventor
Li Zhong
Sergei Zolotukhin
Lakshmanan Govindasamy
Mavis Agbandje-Mckenna
Arun Srivastava
Original Assignee
University Of Florida Research Foundation, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University Of Florida Research Foundation, Inc. filed Critical University Of Florida Research Foundation, Inc.
Publication of PL2191001T3 publication Critical patent/PL2191001T3/pl
Publication of PL2191001T4 publication Critical patent/PL2191001T4/pl

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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    • 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
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    • 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
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    • 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
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/515—Animal cells
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
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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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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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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    • 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
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Manufacturing & Machinery (AREA)
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  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Oncology (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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PL08733161T 2007-04-09 2008-04-08 Kompozycje wektora RAAV mające białka kapsydu zmodyfikowane tyrozyną i sposoby ich zastosowania PL2191001T4 (pl)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US91079807P 2007-04-09 2007-04-09

Publications (2)

Publication Number Publication Date
PL2191001T3 PL2191001T3 (pl) 2017-01-31
PL2191001T4 true PL2191001T4 (pl) 2017-01-31

Family

ID=39684174

Family Applications (1)

Application Number Title Priority Date Filing Date
PL08733161T PL2191001T4 (pl) 2007-04-09 2008-04-08 Kompozycje wektora RAAV mające białka kapsydu zmodyfikowane tyrozyną i sposoby ich zastosowania

Country Status (13)

Country Link
US (11) US8445267B2 (pl)
EP (3) EP2191001B1 (pl)
CA (1) CA2720097C (pl)
CY (1) CY1118228T1 (pl)
DK (2) DK2623605T3 (pl)
ES (3) ES2599632T3 (pl)
HR (1) HRP20161150T1 (pl)
HU (1) HUE030719T2 (pl)
LT (2) LT2191001T (pl)
PL (1) PL2191001T4 (pl)
PT (1) PT2191001T (pl)
SI (1) SI2191001T1 (pl)
WO (1) WO2008124724A1 (pl)

Families Citing this family (145)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2191001T4 (pl) * 2007-04-09 2017-01-31 University Of Florida Research Foundation, Inc. Kompozycje wektora RAAV mające białka kapsydu zmodyfikowane tyrozyną i sposoby ich zastosowania
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
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
CA2762118A1 (en) 2008-05-20 2010-01-28 Eos Neuroscience, 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
US8840881B2 (en) * 2008-08-28 2014-09-23 Aduro Gvax Inc. Methods and compositions for treating prostate cancer or inducing a humoral immune response against prostate cancer
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2011127088A2 (en) 2010-04-05 2011-10-13 Eos Neuroscience, Inc. Methods and compositions for decreasing chronic pain
JP2013533847A (ja) 2010-04-23 2013-08-29 ユニバーシティ オブ マサチューセッツ コレステロール関連障害のａａｖベースの治療
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
ES2605305T3 (es) 2010-04-23 2017-03-13 University Of Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
GB2498325B (en) 2010-10-15 2018-05-02 Eos Neuroscience Inc Engineered human GRM6 enhancer sequence for use in eye therapies
JP5704361B2 (ja) 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
US20140162319A2 (en) * 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
US9677088B2 (en) * 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US20140065110A1 (en) * 2012-08-31 2014-03-06 The Regents Of The University Of California Genetically modified msc and therapeutic methods
DK2900686T3 (da) 2012-09-28 2020-08-17 Univ North Carolina Chapel Hill Aav-vektorer targeteret til oligodendrocytter
JP6628286B2 (ja) * 2013-05-21 2020-01-08 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
WO2015126972A1 (en) * 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
WO2015134643A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
GB2526339A (en) * 2014-05-21 2015-11-25 Imp Innovations Ltd Lentiviral vectors
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US10370432B2 (en) 2014-10-03 2019-08-06 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
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
SG10202001102XA (en) 2014-11-14 2020-03-30 Voyager Therapeutics Inc Modulatory polynucleotides
CN107109407A (zh) 2014-11-14 2017-08-29 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
WO2016081927A2 (en) 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
WO2016133917A1 (en) 2015-02-16 2016-08-25 University Of Florida Research Foundation Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes
WO2016134337A1 (en) * 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Site-specific integrating recombinant aav vectors for gene therapy and improved production methods
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US11253576B2 (en) 2015-10-22 2022-02-22 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
GB201519086D0 (en) 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
JP6996728B2 (ja) * 2016-01-07 2022-01-17 学校法人自治医科大学 グルコーストランスポーター１発現用アデノ随伴ウイルスベクター
AU2017207917B2 (en) 2016-01-13 2024-03-07 Genzyme Corporation Recombinant AAV vectors expressing osteoprotective genes, including HAS2 and lubricin, useful in the treatment of osteoarthritis and related joint conditions in mammals
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
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
PL3436051T3 (pl) 2016-03-31 2022-02-07 Spark Therapeutics, Inc. W pełni skalowalny sposób kolumnowego wytwarzania raav
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
CR20180589A (es) 2016-05-13 2019-02-27 4D Molecular Therapeutics Inc Cápsulas variantes de virus adenoasociados y métodos de uso de estas
WO2017201121A1 (en) * 2016-05-17 2017-11-23 University Of Florida Research Foundation, Inc. Recombinant aav for gene therapy in lungs
EP3458589A4 (en) 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING MORBUS HUNTINGTON
EP3458588B1 (en) 2016-05-18 2025-10-29 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
WO2018033542A1 (en) 2016-08-15 2018-02-22 Danmarks Tekniske Universitet Method for reducing ammonium and lactate production in cho cells
CA3035522A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10650621B1 (en) 2016-09-13 2020-05-12 Iocurrents, Inc. Interfacing with a vehicular controller area network
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CN110506119A (zh) 2016-10-13 2019-11-26 马萨诸塞大学 Aav衣壳设计
JP7215716B2 (ja) 2017-01-13 2023-01-31 学校法人自治医科大学 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのａａｖベクター
JP7081767B2 (ja) 2017-01-30 2022-06-07 学校法人日本医科大学 アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
EP4218828A3 (en) 2017-09-20 2023-09-27 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
KR102793696B1 (ko) 2017-11-27 2025-04-09 4디 몰레큘러 테라퓨틱스 아이엔씨. 아데노-관련 바이러스 변이체 캡시드 및 혈관 신생을 억제하기 위한 용도
US10806802B2 (en) 2018-01-18 2020-10-20 University Of Guelph Adeno-associated virus particle with mutated capsid and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
SG11202009450SA (en) 2018-04-03 2020-10-29 Stridebio Inc Virus vectors for targeting ophthalmic tissues
WO2019210249A1 (en) * 2018-04-26 2019-10-31 Oregon Health & Science University Methods of aav vector production by modulating deubiquitinating enzyme activity
US20210363192A1 (en) * 2018-04-27 2021-11-25 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
JP2021522840A (ja) * 2018-05-15 2021-09-02 プレジデント アンド フェローズ オブ ハーバード カレッジ 改善された遺伝子送達特性を示すウイルスベクター
EP3831949A4 (en) 2018-07-30 2022-05-04 Gene Therapy Research Institution Co., Ltd. METHODS OF INCREASING GENE EXPRESSION BY AN AAV VECTOR
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
EP3861131A4 (en) 2018-10-01 2022-06-15 Ultragenyx Pharmaceutical Inc. GENE THERAPY FOR THE TREATMENT OF PROPIONIC ACIDosis
GB201821089D0 (en) 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
GB201821082D0 (en) 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
WO2020174369A2 (en) 2019-02-25 2020-09-03 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
CN113677801A (zh) 2019-02-25 2021-11-19 诺华股份有限公司 治疗bietti晶体营养不良的组合物和方法
SG11202110146VA (en) 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
GB201906052D0 (en) 2019-04-30 2019-06-12 Int Centre For Genetic Engineering And Biotechnology Proteins with cardioprotective activity
CA3145309A1 (en) 2019-07-10 2021-01-14 Locanabio, Inc. Rna-targeting knockdown and replacement compositions and methods for use
JP7371954B2 (ja) 2019-07-12 2023-10-31 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
JP2022550435A (ja) 2019-10-04 2022-12-01 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 組換えａａｖの改善された治療的使用のための方法
US20230392163A1 (en) 2019-10-17 2023-12-07 Ginkgo Bioworks Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
EP4097121A1 (en) 2020-01-29 2022-12-07 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
IL295529A (en) 2020-02-13 2022-10-01 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
MX2022009982A (es) 2020-02-14 2022-09-12 Ultragenyx Pharmaceutical Inc Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5.
JP2023518415A (ja) 2020-03-19 2023-05-01 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 組換えａａｖにおいてｃａｐおよびｒｅｐ配列の逆パッケージングを低減するための組成物および方法
EP4127189A1 (en) 2020-03-31 2023-02-08 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
CN116745409A (zh) 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
EP4228610A1 (en) 2020-10-13 2023-08-23 Kriya Therapeutics, Inc. Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
CN119585299A (zh) 2020-10-16 2025-03-07 吉尔奥科普治疗有限公司 编码抗vegf实体和补体负调节剂的核酸及其用于年龄相关性黄斑变性的治疗的用途
CA3200588A1 (en) 2020-12-01 2022-06-09 David A. Nelles Rna-targeting compositions and methods for treating myotonic dystrophy type 1
KR20230127221A9 (ko) 2020-12-01 2024-12-06 로카나바이오 인크. Cag 반복 질환을 치료하기 위한 rna 표적화 조성물 및 방법
JP2024503877A (ja) * 2021-01-20 2024-01-29 ザ リサーチ ファウンデイション フォー ザ ステイト ユニバーシティー オブ ニューヨーク 免疫寛容誘導のための組成物及び遺伝子治療におけるその使用
WO2022221278A1 (en) 2021-04-12 2022-10-20 Locanabio, Inc. Compositions and methods comprising hybrid promoters
CN113121655B (zh) * 2021-04-19 2021-11-19 上海信致医药科技有限公司 眼部和肌肉特异靶向型腺相关病毒载体及其应用
EP4328313A4 (en) 2021-04-21 2025-06-25 Jichi Medical University Adeno-associated virus virion for treating ornithine transcarbamylase deficiency
EP4367251A1 (en) 2021-07-08 2024-05-15 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
US20250019721A1 (en) 2021-08-11 2025-01-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
CA3229580A1 (en) * 2021-08-20 2023-02-23 Anna Nikolaevna STRELKOVA Method for producing modified adeno-associated virus capsid
WO2023101281A1 (ko) * 2021-11-30 2023-06-08 연세대학교 산학협력단 아데노 관련 바이러스 캡시드 단백질의 돌연변이체
KR102830431B1 (ko) * 2021-11-30 2025-07-08 주식회사 글루진테라퓨틱스 아데노 관련 바이러스 캡시드 단백질의 돌연변이체
EP4446419A1 (en) 2021-12-06 2024-10-16 Jichi Medical University Recombinant adeno-associated virus vector for treatment of iron-accumulating neurodegenerative diseases
WO2023154807A2 (en) 2022-02-09 2023-08-17 Locanabio, Inc. Compositions and methods for modulating pre-mrna splicing
EP4486888A1 (en) 2022-03-04 2025-01-08 Locanabio, Inc. Compositions and methods comprising engineered small nuclear rna (snrna)
JP2025509328A (ja) 2022-03-07 2025-04-11 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 改良されたバッチａａｖ産生システムおよび方法
WO2023205637A1 (en) 2022-04-18 2023-10-26 Locanabio, Inc. Rna-targeting compositions and methods for treating c9/orf72 diseases
EP4543903A2 (en) * 2022-06-22 2025-04-30 Synvivia, Inc. Methods of selection and use of engineered cyclic peptides for enhancing viral production
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies
IL320277A (en) 2022-10-18 2025-06-01 Regeneron Pharma Compositions and methods including programmable snRNAs for RNA editing
CN120548368A (zh) 2022-12-01 2025-08-26 再生元制药公司 用于正确包装重复元件的腺相关病毒载体
IL321968A (en) 2023-02-02 2025-09-01 Quethera Ltd Recombinant adeno-associated virus vector
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2025072530A2 (en) 2023-09-26 2025-04-03 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating dmd
WO2025090628A1 (en) 2023-10-23 2025-05-01 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies
WO2025126158A2 (en) 2023-12-15 2025-06-19 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) targeting sod1
WO2025155722A1 (en) 2024-01-16 2025-07-24 Astellas Gene Therapies, Inc. Muscle selective hybrid regulatory combinations and methods of use thereof for the treatment of myotonic dystrophy type 1
WO2025160364A2 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for the treatment of pompe disease
WO2025253097A1 (en) 2024-06-03 2025-12-11 Forcefield Therapeutics Limited Proteins with cardiopreservation activity

Family Cites Families (108)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US102A (en) 1861-08-13 Whole
US106A (en) 1915-08-24 Specipication
US103A (en) 1836-12-10 Process
US104A (en) 1836-12-10 Revolving hokse-rake
US105A (en) 1836-12-15 knight
EP0003048B2 (de) 1978-01-05 1983-08-24 Bayer Ag Formmassen auf der Grundlage ungesättigter Polyester, Verfahren zu ihrer Herstellung und ihre Verwendung
US4216209A (en) 1979-03-19 1980-08-05 American Home Products Corporation Tripeptide angiotensin converting enzyme inhibitors
US4554101A (en) 1981-01-09 1985-11-19 New York Blood Center, Inc. Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity
US4883750A (en) 1984-12-13 1989-11-28 Applied Biosystems, Inc. Detection of specific sequences in nucleic acids
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4800159A (en) 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US5037746A (en) 1986-12-03 1991-08-06 University Patents, Inc. RNA ribozyme polymerases, and methods
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5116742A (en) 1986-12-03 1992-05-26 University Patents, Inc. RNA ribozyme restriction endoribonucleases and methods
AU622104B2 (en) 1987-03-11 1992-04-02 Sangtec Molecular Diagnostics Ab Method of assaying of nucleic acids, a reagent combination and kit therefore
IL86724A (en) 1987-06-19 1995-01-24 Siska Diagnostics Inc Methods and kits for amplification and testing of nucleic acid sequences
AU622426B2 (en) 1987-12-11 1992-04-09 Abbott Laboratories Assay using template-dependent nucleic acid probe reorganization
AU624601B2 (en) 1988-01-21 1992-06-18 Genentech Inc. Amplification and detection of nucleic acid sequences
CA1340807C (en) 1988-02-24 1999-11-02 Lawrence T. Malek Nucleic acid amplification process
US4932207A (en) 1988-12-28 1990-06-12 Sundstrand Corporation Segmented seal plate for a turbine engine
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
US5219727A (en) 1989-08-21 1993-06-15 Hoffmann-Laroche Inc. Quantitation of nucleic acids using the polymerase chain reaction
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
WO1991005041A1 (en) 1989-09-26 1991-04-18 Townes Tim M Erythroid-specific gene expression system
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
JPH03251543A (ja) 1990-02-26 1991-11-11 Unitika Ltd アンジオテンシン変換酵素阻害剤
DE69124274T2 (de) 1990-02-27 1997-08-14 Agency Ind Science Techn Oligopeptide, sie enthaltende pharmazeutische und Futterzusammensetzung und Benützung von Oligopeptiden
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
IL98546A (en) 1990-06-22 1996-05-14 Fisons Plc Ace inhibitor ester derivatives their preparation and pharmaceutical compositions containing them
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5145684A (en) 1991-01-25 1992-09-08 Sterling Drug Inc. Surface modified drug nanoparticles
US5455166A (en) 1991-01-31 1995-10-03 Becton, Dickinson And Company Strand displacement amplification
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
PT620849E (pt) 1992-01-07 2003-11-28 Elan Pharm Inc Modelos animais transgenicos para a doenca de alzheimer
US5552397A (en) 1992-05-18 1996-09-03 E. R. Squibb & Sons, Inc. Substituted azepinone dual inhibitors of angiotensin converting enzyme and neutral exdopeptidase
US5646020A (en) 1992-05-14 1997-07-08 Ribozyme Pharmaceuticals, Inc. Hammerhead ribozymes for preferred targets
WO1993023011A1 (en) 1992-05-18 1993-11-25 Minnesota Mining And Manufacturing Company Transmucosal drug delivery device
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
US5297721A (en) 1992-11-19 1994-03-29 Fry's Metals, Inc. No-clean soldering flux and method using the same
US5639655A (en) 1993-01-19 1997-06-17 Ribozyme Pharmaceuticals, Inc. PML-RARA targeted ribozymes
US5279721A (en) 1993-04-22 1994-01-18 Peter Schmid Apparatus and method for an automated electrophoresis system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5639940A (en) 1994-03-03 1997-06-17 Pharmaceutical Proteins Ltd. Production of fibrinogen in transgenic animals
US5648211A (en) 1994-04-18 1997-07-15 Becton, Dickinson And Company Strand displacement amplification using thermophilic enzymes
US5789655A (en) 1994-05-13 1998-08-04 The Regents Of The University Of California Transgenic animals expressing artificial epitope-tagged proteins
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
US5580579A (en) 1995-02-15 1996-12-03 Nano Systems L.L.C. Site-specific adhesion within the GI tract using nanoparticles stabilized by high molecular weight, linear poly (ethylene oxide) polymers
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5646031A (en) 1995-05-16 1997-07-08 Northern Illinois University SArMV and sCYMVI hairpin ribozymes
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
US5863736A (en) 1997-05-23 1999-01-26 Becton, Dickinson And Company Method, apparatus and computer program products for determining quantities of nucleic acid sequences in samples
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US7052692B1 (en) * 1997-09-02 2006-05-30 Advanced Research & Technology Institute Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
EP1285078A2 (en) 2000-04-28 2003-02-26 The Trustees of The University of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
EP1419245A4 (en) * 2001-07-13 2006-04-05 Univ Iowa Res Found ADENO-ASSOCIATED PSEUDOTYPE VIRUSES AND USES THEREOF
MX359371B (es) * 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
DK1463805T3 (en) * 2001-12-17 2015-01-19 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing these as well as their uses
EP2036985A1 (en) 2002-04-09 2009-03-18 Nationwide Children's Hospital, Inc. Antibody gene transfer and recombinant AAV therefor
AU2003221733A1 (en) 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
DE60323078D1 (de) 2002-05-01 2008-10-02 Univ Florida Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
PL3235827T3 (pl) 2003-06-19 2021-07-05 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania
EP2345731B1 (en) * 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US8999678B2 (en) 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
US20090215870A1 (en) 2005-04-29 2009-08-27 Terwilliger Ernest F Compositions and methods for targeting of viral vectors
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
PL2191001T4 (pl) 2007-04-09 2017-01-31 University Of Florida Research Foundation, Inc. Kompozycje wektora RAAV mające białka kapsydu zmodyfikowane tyrozyną i sposoby ich zastosowania
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
CA2762118A1 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
WO2009154452A1 (en) 2008-06-17 2009-12-23 Amsterdam Molecular Therapeutics B.V. Parvoviral capsid with incorporated Gly-Ala repeat region
US8312249B1 (en) 2008-10-10 2012-11-13 Apple Inc. Dynamic trampoline and structured code generation in a signed code environment
FR2957821B1 (fr) 2010-03-24 2014-08-29 Inst Francais Du Petrole Nouvelle zone de regeneration du catalyseur divisee en secteurs pour unites catalytiques regeneratives
ES2605305T3 (es) 2010-04-23 2017-03-13 University Of Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
JP5704361B2 (ja) 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
DK2673289T3 (da) 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf
MX359518B (es) 2012-04-18 2018-10-01 Childrens Hospital Philadelphia Composición y métodos para una transferencia génica altamente eficiente utilizando variantes de cápsides de aav.
US20140162319A2 (en) 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
US12358954B2 (en) * 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
CN103060331B (zh) 2012-12-05 2014-10-08 南京医科大学第一附属医院 介导膜稳定cd40l基因的衣壳蛋白突变的双链重组腺相关病毒制备方法与应用
AU2014251099B2 (en) * 2013-04-08 2019-01-17 The University Of Kansas Chimeric adeno-associated virus/ bocavirus parvovirus vector
JP6628286B2 (ja) 2013-05-21 2020-01-08 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法
WO2015134643A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
CA3234028A1 (en) * 2014-03-11 2015-09-17 Wayne State University A modified mglur6 promoter and methods of use
WO2016081927A2 (en) 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
US20180030096A1 (en) * 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
WO2016133917A1 (en) 2015-02-16 2016-08-25 University Of Florida Research Foundation Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes
US20180135074A1 (en) 2015-02-19 2018-05-17 University Of Florida Research Foundation, Incorporated Recombinant aav vectors for gene therapy of human hematopoietic disorders
US11266748B2 (en) 2015-07-02 2022-03-08 University Of Florida Research Foundation, Incorporated Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
US20190000943A1 (en) 2015-08-13 2019-01-03 Aavet Therapeutics, Llc Aav6 vectors for immunotherapy
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
US20240181083A1 (en) 2021-04-23 2024-06-06 University Of Florida Research Foundation, Incorporated Aavrh74 vectors for gene therapy of muscular dystrophies

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