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PT3684423T - Cápsides variantes de vírus adeno-associado e métodos de utilização das mesmas - Google Patents

Cápsides variantes de vírus adeno-associado e métodos de utilização das mesmas

Info

Publication number
PT3684423T
PT3684423T PT188592414T PT18859241T PT3684423T PT 3684423 T PT3684423 T PT 3684423T PT 188592414 T PT188592414 T PT 188592414T PT 18859241 T PT18859241 T PT 18859241T PT 3684423 T PT3684423 T PT 3684423T
Authority
PT
Portugal
Prior art keywords
adeno
methods
associated virus
virus variant
variant capsids
Prior art date
Application number
PT188592414T
Other languages
English (en)
Original Assignee
4D Molecular Therapeutics 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 4D Molecular Therapeutics Inc filed Critical 4D Molecular Therapeutics Inc
Publication of PT3684423T publication Critical patent/PT3684423T/pt

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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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1719—Muscle proteins, e.g. myosin or actin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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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
    • 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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
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    • C12Y116/00—Oxidoreductases oxidizing metal ions (1.16)
    • C12Y116/03—Oxidoreductases oxidizing metal ions (1.16) with oxygen as acceptor (1.16.3)
    • C12Y116/03001—Ferroxidase (1.16.3.1), i.e. ceruloplasmin
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • 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
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    • C12N2750/00011—Details
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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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PT188592414T 2017-09-20 2018-09-19 Cápsides variantes de vírus adeno-associado e métodos de utilização das mesmas PT3684423T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762560901P 2017-09-20 2017-09-20

Publications (1)

Publication Number Publication Date
PT3684423T true PT3684423T (pt) 2023-06-09

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PT188592414T PT3684423T (pt) 2017-09-20 2018-09-19 Cápsides variantes de vírus adeno-associado e métodos de utilização das mesmas

Country Status (25)

Country Link
US (3) US11118192B2 (pt)
EP (2) EP3684423B1 (pt)
JP (1) JP6825167B2 (pt)
KR (3) KR102743219B1 (pt)
CN (1) CN111836649A (pt)
AU (1) AU2018337833B2 (pt)
BR (1) BR112020005436B1 (pt)
CA (1) CA3076348A1 (pt)
CL (1) CL2020000728A1 (pt)
CO (1) CO2020004738A2 (pt)
CR (1) CR20200165A (pt)
DK (1) DK3684423T5 (pt)
ES (1) ES2948902T3 (pt)
FI (1) FI3684423T3 (pt)
IL (1) IL273261B1 (pt)
MX (1) MX2020003176A (pt)
NZ (1) NZ763018A (pt)
PE (1) PE20201264A1 (pt)
PH (1) PH12020550094A1 (pt)
PT (1) PT3684423T (pt)
RU (1) RU2770922C2 (pt)
SG (1) SG11202002276VA (pt)
UA (1) UA127455C2 (pt)
WO (1) WO2019060454A2 (pt)
ZA (1) ZA202001679B (pt)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7348844B2 (ja) 2017-06-07 2023-09-21 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 内部移行酵素のための組成物および方法
KR102743219B1 (ko) 2017-09-20 2024-12-17 4디 몰레큘러 테라퓨틱스 아이엔씨. 아데노-관련 바이러스 변이체 캡시드 및 그 용도
FI3717636T3 (fi) 2017-11-27 2023-06-01 4D Molecular Therapeutics Inc Adenoassosioidun viruksen kapsidivariantteja ja käyttö angiogeneesin estämisessä
CN113164556A (zh) 2018-08-30 2021-07-23 特纳亚治疗股份有限公司 用心肌蛋白和ascl1进行的心脏细胞重编程
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
CN110437317B (zh) * 2019-01-30 2023-05-02 上海科技大学 具有变异衣壳蛋白的腺相关病毒及其用途
WO2020198233A1 (en) * 2019-03-25 2020-10-01 The Regents Of The University Of California Methods of treating tnni3-mediated cardiomyopathy
EP3947700A4 (en) * 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. ADENO-ASSOCIATED VIRUS WITH MANIPULATED CAPSID
WO2021007515A1 (en) 2019-07-11 2021-01-14 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
JP2022549140A (ja) * 2019-09-20 2022-11-24 ユーシーエル ビジネス リミテッド 遺伝子療法組成物および不整脈源性右心室心筋症の治療
KR20220105158A (ko) * 2019-10-23 2022-07-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이식유전자 발현의 drg-특이적 감소를 위한 조성물
BR112022009864A2 (pt) * 2019-11-22 2022-08-02 Childrens Hospital Philadelphia Variantes do vetor viral adenoassociado
BR112022015979A2 (pt) 2020-02-13 2022-10-11 Tenaya Therapeutics Inc Vetores de terapia genética para tratamento de doenças cardíacas
US20230108316A1 (en) * 2020-04-08 2023-04-06 University Of Utah Research Foundation Methods for rehabilitating heart failure using gene therapy
WO2021222094A1 (en) * 2020-04-27 2021-11-04 4D Molecular Therapeutics Inc. Codon optimized gla genes and uses thereof
AU2021273447A1 (en) 2020-05-13 2022-12-08 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
US11900272B2 (en) 2020-05-13 2024-02-13 Factset Research System Inc. Method and system for mapping labels in standardized tables using machine learning
EP4149519A4 (en) * 2020-05-14 2024-06-12 The Trustees of The University of Pennsylvania COMPOSITIONS USEFUL IN THE TREATMENT OF PUMP DISEASE
US20230226221A1 (en) * 2020-06-30 2023-07-20 Children's National Medical Center Use of recombinant human acid sphingomyelinase to improve skeletal myofiber repair
US20230313186A1 (en) * 2020-07-24 2023-10-05 The Trustees Of The University Of Pennsylvania Trophin genome editing for treating duchenne muscular dystrophy (dmd)
CN112194706B (zh) * 2020-09-30 2022-03-08 广州派真生物技术有限公司 腺相关病毒突变体及其应用
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
WO2022103766A2 (en) * 2020-11-10 2022-05-19 Avirmax, Inc. Engineered viral capsids and methods of use
CN112725342B (zh) * 2021-02-02 2024-05-17 中国科学院深圳先进技术研究院 一种启动子pCALM2及其应用
US20230062110A1 (en) * 2021-03-26 2023-03-02 Adverum Biotechnologies, Inc. Intravitreal dosing for delivery of polynucleotides to retinal cones
EP4322974A2 (en) * 2021-04-13 2024-02-21 Capsida, Inc. Adeno-associated virus compositions having increased brain enrichment
CN113121653B (zh) * 2021-04-19 2021-11-19 上海信致医药科技有限公司 肌肉和视网膜特异性的新型腺相关病毒衣壳蛋白
EP4347621A1 (en) * 2021-06-03 2024-04-10 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2023005906A1 (zh) * 2021-07-30 2023-02-02 上海信致医药科技有限公司 新型腺相关病毒血清型介导的血管生成抑制剂及其应用
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
EP4392570A2 (en) * 2021-08-25 2024-07-03 CANbridge Pharmaceuticals, Inc. Aav particles comprising liver-tropic capsid protein and acid alpha-glucosidase and use to treat pompe disease
AU2022358779A1 (en) 2021-10-08 2024-04-18 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2024064782A2 (en) * 2022-09-21 2024-03-28 Rejuvenate Bio Adeno-associated virus capsids
WO2024073735A2 (en) * 2022-09-30 2024-04-04 University Of Massachusetts Oligonucleotides targeting s6k1
CN115960177B (zh) * 2022-10-09 2023-07-07 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN115838399B (zh) * 2022-12-08 2023-08-11 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024130070A2 (en) * 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
CN115925819B (zh) * 2022-12-30 2023-10-13 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN116041443B (zh) * 2022-12-30 2023-09-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2024206831A1 (en) * 2023-03-31 2024-10-03 Duke University Compositions comprising brain-tropic aavs and methods of use thereof

Family Cites Families (93)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US7323179B2 (en) 1997-12-19 2008-01-29 Naomi Balaban Methods and compositions for the treatment and prevention of Staphylococcus and other bacterial infections
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
AU762220B2 (en) 1998-05-28 2003-06-19 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The 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
PT1127150E (pt) 1998-11-05 2007-08-22 Univ Pennsylvania ''sequências de ácido nucleico do vírus adeno associado do serotipo 1, vectores e células hospedeiras que as contêm''
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
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
JP2003516124A (ja) 1999-10-15 2003-05-13 ユニバーシティー オブ マサチューセッツ 標的とした遺伝的干渉の手段としてのｒｎａ干渉経路遺伝子
ES2745378T3 (es) 2000-03-30 2020-03-02 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de RNA
DE60141014D1 (de) 2000-05-23 2010-02-25 Univ Rochester Verfharen zur herstellung von herpes-simplex-virus-amplikons, die resultierenden amplikons und ihre verwendung
CA2410947A1 (en) 2000-05-30 2001-12-06 Johnson & Johnson Research Pty Limited Methods for mediating gene suppresion by using factors that enhance rnai
EP1309706A2 (en) 2000-08-19 2003-05-14 Axordia Limited Modulation of stem cell differentiation
WO2002029858A2 (en) 2000-09-29 2002-04-11 Infineon Technologies North America Corp. Deep trench etching method to reduce/eliminate formation of black silicon
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
NZ578982A (en) 2001-11-13 2011-03-31 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
ES2717377T3 (es) 2001-12-17 2019-06-20 Univ Pennsylvania Secuencias de serotipo 8 de virus adeno-asociado (AAV), vectores que las contienen y usos de las mismas
PT2573170T (pt) 2001-12-17 2018-03-26 Univ Pennsylvania Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações
JP2005512569A (ja) 2001-12-21 2005-05-12 メディジーン・アクチェンゲゼルシャフト 目的とする細胞指向性をもつウイルスクローンの同定に有用な修飾した構造遺伝子またはキャプシド修飾した粒子のライブラリー
AU2003253595A1 (en) 2002-04-05 2003-11-03 The Children's Hospital Of Philadelphia Methods for the production of chimeric adeno-associated virus (aav) vectors, compositions of chimeric aav vectors, and methods of use thereof
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
DE60323078D1 (de) 2002-05-01 2008-10-02 Univ Florida Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine
US20040023390A1 (en) 2002-08-05 2004-02-05 Davidson Beverly L. SiRNA-mediated gene silencing with viral vectors
WO2004020600A2 (en) 2002-08-28 2004-03-11 University Of Florida Modified aav
US20070172460A1 (en) * 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
US7186522B2 (en) 2003-03-31 2007-03-06 Cytyc Corporation Papanicolau staining process
US20060286545A1 (en) 2003-05-23 2006-12-21 Mount Sinai School Of Medicine Of New York University Viral vectors with improved properties
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US8071028B2 (en) 2003-06-12 2011-12-06 Abbott Diabetes Care Inc. Method and apparatus for providing power management in data communication systems
DK2657248T3 (en) 2003-06-19 2017-06-26 Genzyme Corp AAV virions with reduced immunoreactivity and applications thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
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
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 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
ATE465173T1 (de) 2004-09-24 2010-05-15 Intercell Ag Verändertes vp-1capsidprotein von parvovirus b19
CA2591544A1 (en) 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Chimeric vectors
CN117363655A (zh) 2005-04-07 2024-01-09 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
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
ES2410133T3 (es) 2005-10-20 2013-07-01 Uniqure Ip B.V. Vectores de VAA mejorados producidos en células de insecto.
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
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
EP2623605B1 (en) 2007-04-09 2018-11-28 University of Florida Research Foundation, Inc. RAAV vector compositions having tyrosine-modified capsid proteins and methods for use
WO2008124015A1 (en) 2007-04-09 2008-10-16 The Regents Of The University Of California Methods for purifying adeno-associated virus virions
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
CA3002933A1 (en) 2007-07-14 2009-01-22 Beverly L. Davidson Methods and compositions for treating brain diseases
US20090215879A1 (en) 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
EP2297185A1 (en) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Parvoviral capsid with incorporated gly-ala repeat region
JP2010002479A (ja) 2008-06-18 2010-01-07 Crossfor:Kk メガネレンズ用装飾体およびメガネ用装飾体装着具
WO2010031865A1 (en) 2008-09-19 2010-03-25 Charitē Universitätsmedizin Berlin Identification and characterisation of recombinant viral gene therapy vectors
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9133479B2 (en) 2009-06-03 2015-09-15 Cedars-Sinai Medical Center Effective vector platform for gene transfer and gene therapy
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
US8299295B2 (en) 2009-10-15 2012-10-30 Johnson Matthey Public Limited Company Polymorphs of bromfenac sodium and methods for preparing bromfenac sodium polymorphs
US8263396B2 (en) 2010-04-01 2012-09-11 Weidong Xiao Methods and compositions for the production of recombinant virus vectors
JP5963743B2 (ja) 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Ｃｎｓターゲティングａａｖベクターおよびその使用方法
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
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
US10415056B2 (en) 2010-11-10 2019-09-17 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
DK2673289T3 (da) 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf
WO2012112578A2 (en) 2011-02-14 2012-08-23 The Children's Hospital Of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
CA2826316C (en) 2011-02-17 2021-04-27 The Trustees Of The University Of Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
PL3693025T3 (pl) * 2011-04-22 2022-03-14 The Regents Of The University Of California Wiriony wirusa towarzyszącego adenowirusom z różnymi kapsydami i sposoby ich zastosowania
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
US20140359799A1 (en) 2011-12-23 2014-12-04 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
CN104520428B (zh) 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
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.
EP2660325A3 (en) 2012-05-02 2014-02-12 Christian Medical College AAV vectors and corresponding nucleotide sequences and methods
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
WO2013174760A1 (en) 2012-05-25 2013-11-28 Deutsches Krebsforschungszentrum Optimized aav-vectors for high transduction rates in dendritic cells
EP2692731A1 (en) 2012-07-31 2014-02-05 Paul-Ehrlich-Institut Bundesamt für Sera und Impfstoffe Recombinant Adeno-Associated virus (AAV) vector particles displaying high-affinity ligands for cell-type specific gene delivery
US9636370B2 (en) 2012-09-28 2017-05-02 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
JP6363958B2 (ja) 2012-12-25 2018-07-25 タカラバイオ株式会社 Ａａｖ変異体
ES2741502T3 (es) 2013-02-08 2020-02-11 Univ Pennsylvania Cápside de AAV8 modificada para la transferencia génica para terapias de retina
WO2014160092A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
JP6600624B2 (ja) 2013-05-31 2019-10-30 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 アデノ随伴ウイルス変異体及びその使用方法
EP4331590A3 (en) * 2013-10-29 2024-04-17 President and Fellows of Harvard College Nuclear factor erythroid 2-like 2 (nrf2) for use in treatment of age-related macular degeneration
KR102537394B1 (ko) * 2014-03-17 2023-05-30 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
US11021519B2 (en) * 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
MX2017012097A (es) 2015-03-24 2018-06-15 Univ California Variantes de virus adenoasociado y métodos de uso de estas.
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
EP4209501A1 (en) * 2016-05-13 2023-07-12 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
CA3029833A1 (en) * 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
KR102743219B1 (ko) * 2017-09-20 2024-12-17 4디 몰레큘러 테라퓨틱스 아이엔씨. 아데노-관련 바이러스 변이체 캡시드 및 그 용도
WO2021222094A1 (en) * 2020-04-27 2021-11-04 4D Molecular Therapeutics Inc. Codon optimized gla genes and uses thereof

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