JP6602788B2 - アデノ随伴ウイルスベクター - Google Patents

アデノ随伴ウイルスベクター Download PDF

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Publication number
JP6602788B2
JP6602788B2 JP2016569022A JP2016569022A JP6602788B2 JP 6602788 B2 JP6602788 B2 JP 6602788B2 JP 2016569022 A JP2016569022 A JP 2016569022A JP 2016569022 A JP2016569022 A JP 2016569022A JP 6602788 B2 JP6602788 B2 JP 6602788B2
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capsid protein
variant
aav
sequence
wild type
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Japanese (ja)
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JP2017506521A5 (cg-RX-API-DMAC7.html
JP2017506521A (ja
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マイケル リンデン ラルフ
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Kings College London
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Kings College London
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    • 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
    • C12N15/86—Viral vectors
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • 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
    • 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
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2016569022A 2014-02-17 2015-02-17 アデノ随伴ウイルスベクター Active JP6602788B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2019186201A JP6942166B2 (ja) 2014-02-17 2019-10-09 アデノ随伴ウイルスベクター

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461940639P 2014-02-17 2014-02-17
US61/940,639 2014-02-17
GBGB1403684.2A GB201403684D0 (en) 2014-03-03 2014-03-03 Vector
GB1403684.2 2014-03-03
PCT/EP2015/053335 WO2015121501A1 (en) 2014-02-17 2015-02-17 Adeno-associated virus vector

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019186201A Division JP6942166B2 (ja) 2014-02-17 2019-10-09 アデノ随伴ウイルスベクター

Publications (3)

Publication Number Publication Date
JP2017506521A JP2017506521A (ja) 2017-03-09
JP2017506521A5 JP2017506521A5 (cg-RX-API-DMAC7.html) 2018-03-29
JP6602788B2 true JP6602788B2 (ja) 2019-11-06

Family

ID=50490674

Family Applications (4)

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JP2016569022A Active JP6602788B2 (ja) 2014-02-17 2015-02-17 アデノ随伴ウイルスベクター
JP2019186201A Active JP6942166B2 (ja) 2014-02-17 2019-10-09 アデノ随伴ウイルスベクター
JP2021086867A Active JP7309780B2 (ja) 2014-02-17 2021-05-24 アデノ随伴ウイルスベクター
JP2023109822A Active JP7612767B2 (ja) 2014-02-17 2023-07-04 アデノ随伴ウイルスベクター

Family Applications After (3)

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JP2019186201A Active JP6942166B2 (ja) 2014-02-17 2019-10-09 アデノ随伴ウイルスベクター
JP2021086867A Active JP7309780B2 (ja) 2014-02-17 2021-05-24 アデノ随伴ウイルスベクター
JP2023109822A Active JP7612767B2 (ja) 2014-02-17 2023-07-04 アデノ随伴ウイルスベクター

Country Status (20)

Country Link
US (4) US20180135076A1 (cg-RX-API-DMAC7.html)
EP (2) EP3597760A3 (cg-RX-API-DMAC7.html)
JP (4) JP6602788B2 (cg-RX-API-DMAC7.html)
KR (1) KR102082032B1 (cg-RX-API-DMAC7.html)
CN (1) CN106062200B (cg-RX-API-DMAC7.html)
AU (3) AU2015216847B2 (cg-RX-API-DMAC7.html)
BR (1) BR112016018598A2 (cg-RX-API-DMAC7.html)
CA (2) CA2939612C (cg-RX-API-DMAC7.html)
DK (1) DK3108000T3 (cg-RX-API-DMAC7.html)
ES (1) ES2749235T3 (cg-RX-API-DMAC7.html)
GB (1) GB201403684D0 (cg-RX-API-DMAC7.html)
HU (1) HUE045801T2 (cg-RX-API-DMAC7.html)
IL (2) IL247332B (cg-RX-API-DMAC7.html)
MX (1) MX372992B (cg-RX-API-DMAC7.html)
PL (1) PL3108000T3 (cg-RX-API-DMAC7.html)
PT (1) PT3108000T (cg-RX-API-DMAC7.html)
RU (1) RU2743382C2 (cg-RX-API-DMAC7.html)
SG (1) SG11201606365TA (cg-RX-API-DMAC7.html)
SI (1) SI3108000T1 (cg-RX-API-DMAC7.html)
WO (1) WO2015121501A1 (cg-RX-API-DMAC7.html)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020022476A (ja) * 2014-02-17 2020-02-13 キングス カレッジ ロンドンＫｉｎｇｓ Ｃｏｌｌｅｇｅ Ｌｏｎｄｏｎ アデノ随伴ウイルスベクター

Families Citing this family (195)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
US10494645B2 (en) 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
EP4410805A3 (en) 2014-03-18 2024-11-27 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
EP3134522B1 (en) 2014-04-25 2021-10-06 University of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
PE20170260A1 (es) * 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
DK3218386T3 (da) 2014-11-14 2021-06-07 Voyager Therapeutics Inc Modulatorisk polynukleotid
WO2016086227A2 (en) * 2014-11-26 2016-06-02 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US11046955B2 (en) 2015-04-24 2021-06-29 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
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
KR20210005994A (ko) 2015-11-05 2021-01-15 뱀부 테라퓨틱스 인코포레이티드 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES
AU2016366549B2 (en) * 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
CN109072254A (zh) * 2015-12-14 2018-12-21 宾夕法尼亚州大学信托人 用于治疗脊髓性肌萎缩的腺相关病毒载体
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
EP3413928B1 (en) 2016-02-12 2022-04-20 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
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
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
CN107287238B (zh) * 2016-04-11 2020-10-16 厦门继景生物技术有限责任公司 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物
AU2017248731B2 (en) 2016-04-15 2024-08-29 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
WO2018002812A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
WO2018002762A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
ES2989940T3 (es) 2016-06-29 2024-11-28 Crispr Therapeutics Ag Materiales y métodos para el tratamiento de la ataxia de Friedreich y otros trastornos relacionados
EP3481857A1 (en) 2016-07-06 2019-05-15 Crispr Therapeutics AG Materials and methods for treatment of pain related disorders
AU2017292169B2 (en) 2016-07-06 2021-12-23 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
US11458211B2 (en) 2016-07-12 2022-10-04 The University Of Manchester Gene therapy
MX2019001276A (es) 2016-07-29 2019-06-13 Univ California Viriones de virus adenoasociados con cápside variante y métodos para su uso.
EP3500676A4 (en) * 2016-08-16 2020-07-01 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR TRANSFERRING TARGETED GENES
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
EP3518985A4 (en) * 2016-09-29 2020-08-05 University of Florida Research Foundation, Incorporated AAVRH.10 VARIANTS WITH HOST ANTIBODY EXHAUST CAPACITIES AND MODIFIED TISSUE TARGETING PROPERTIES
WO2018071831A1 (en) * 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
EP3528785A4 (en) * 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. MODIFIED AAV CASPIDS AND USES THEREOF
CA3042689A1 (en) 2016-11-04 2018-07-12 Baxalta Incorporated Adeno-associated virus formulations
JP7237843B2 (ja) 2017-02-21 2023-03-13 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 修飾されたａａｖキャプシドタンパク質およびその使用
US20200040061A1 (en) 2017-02-22 2020-02-06 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
US11407997B2 (en) 2017-02-22 2022-08-09 Crispr Therapeutics Ag Materials and methods for treatment of primary hyperoxaluria type 1 (PH1) and other alanine-glyoxylate aminotransferase (AGXT) gene related conditions or disorders
EP3585900B1 (en) 2017-02-22 2022-12-21 CRISPR Therapeutics AG Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
US11559588B2 (en) 2017-02-22 2023-01-24 Crispr Therapeutics Ag Materials and methods for treatment of Spinocerebellar Ataxia Type 1 (SCA1) and other Spinocerebellar Ataxia Type 1 Protein (ATXN1) gene related conditions or disorders
CN119351474A (zh) 2017-02-22 2025-01-24 克里斯珀医疗股份公司 用于基因编辑的组合物和方法
UY37679A (es) 2017-04-14 2018-11-30 Regenxbio Inc Tratamiento de mucopolisacaridosis ii con iduronato-2-sulfatasa (ids) humana recombinante producida por células neurales o gliales humanas
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119491003A (zh) 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
WO2018208972A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
JP7330899B2 (ja) 2017-05-10 2023-08-22 マサチューセッツ アイ アンド イヤー インファーマリー ウイルスのアセンブリ活性化タンパク質（ａａｐ）依存性を改変する方法および組成物
CN110831611A (zh) * 2017-05-10 2020-02-21 马萨诸塞眼科耳科诊所 用于修饰病毒的组装活化蛋白(aap)依赖性的方法和组合物
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
AU2018290954B2 (en) * 2017-06-30 2023-07-13 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
TWI722310B (zh) * 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法
US20190085358A1 (en) * 2017-08-25 2019-03-21 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
CA3059995A1 (en) 2017-08-28 2019-03-07 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
EP3684937A4 (en) 2017-09-22 2021-06-02 University of Massachusetts NEW DUAL SOD1 EXPRESSION VECTORS AND RELATED USES
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
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
AU2018352236B2 (en) 2017-10-16 2025-04-10 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
EP4509527A3 (en) * 2017-10-18 2025-04-30 REGENXBIO Inc. Post-translationally modified fully human antibody therapeutic agents
JP7402163B2 (ja) 2017-12-21 2023-12-20 クリスパー セラピューティクス アーゲー ２ａ型アッシャー症候群の処置のための材料および方法
CA3084633A1 (en) 2017-12-21 2019-06-27 Crispr Therapeutics Ag Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
IL277270B2 (en) * 2018-03-16 2025-05-01 Res Inst Nationwide Childrens Hospital Increasing tissue-specific gene delivery through capsid modification
CA3095127A1 (en) * 2018-03-29 2019-10-03 Asklepios Biopharmaceutical, Inc. Liver tropic recombinant aav6 vectors that evade neutralization
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
US12091435B2 (en) 2018-04-03 2024-09-17 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
CA3094311A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN108660159B (zh) * 2018-04-12 2020-10-30 四川大学 重组蝙蝠腺相关病毒载体及其用途
KR102051393B1 (ko) 2018-05-04 2019-12-04 (주)지뉴인텍 아데노부속바이러스 기반 유전자 전달용 재조합 벡터 및 이를 이용한 암질환 예방 또는 치료용 조성물
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
AU2019270900A1 (en) * 2018-05-15 2020-10-15 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
CA3108324A1 (en) * 2018-07-31 2020-02-06 Cornell University Gene therapy methods to control organ function
CA3114621A1 (en) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020072683A1 (en) 2018-10-02 2020-04-09 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
EP3880823A4 (en) * 2018-11-16 2022-08-17 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
GB201900702D0 (en) * 2019-01-18 2019-03-06 Univ Bristol Therapy
WO2020150556A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
KR20210126096A (ko) * 2019-02-15 2021-10-19 상가모 테라퓨틱스, 인코포레이티드 재조합 aav를 제조하기 위한 조성물 및 방법
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
KR20210142725A (ko) * 2019-03-28 2021-11-25 오피노 엘엘씨 코어 페이징 처리
KR20210148232A (ko) 2019-04-01 2021-12-07 테나야 테라퓨틱스, 인코포레이티드 조작된 캡시드를 갖는 아데노-연관 바이러스
CN114127088B (zh) * 2019-04-23 2024-05-14 国家医疗保健研究所 变体衣壳蛋白和腺相关病毒aav变体及其药物组合物
BR112021017603A2 (pt) 2019-04-24 2021-11-16 Takara Bio Inc Vírus adenoassociado (aav) mutante tendo propriedade de direcionamento para o cérebro
AU2020263392A1 (en) * 2019-04-26 2021-10-28 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV2.5 vector
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
CA3144864A1 (en) 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
EP3990636A1 (en) 2019-06-28 2022-05-04 CRISPR Therapeutics AG Materials and methods for controlling gene editing
AU2020299026A1 (en) * 2019-07-04 2022-01-20 Children's Medical Research Institute Methods and AAV vectors for in vivo transduction
US20220265853A1 (en) * 2019-07-12 2022-08-25 Gene Therapy Research Institution Co., Ltd. Adeno-associated virus virion for gene transfer to human liver
EP3999119B1 (en) * 2019-07-15 2024-09-11 Meiragtx UK II Limited Modified aav capsid proteins for treatment of arthritic disease
JP7785352B2 (ja) * 2019-08-14 2025-12-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 遺伝子治療のためのａａｖカプシドバリアント
CA3151087A1 (en) * 2019-08-29 2021-03-04 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral vectors for crossing the human blood brain barrier
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
EP4041755A4 (en) * 2019-10-10 2023-10-25 Solid Biosciences Inc. MODIFIED AAV CAPSIDS AND USES THEREOF
EP4045664A1 (en) * 2019-10-16 2022-08-24 Wuxi Apptec (Shanghai) Co., Ltd. A novel aav variant
US20230392163A1 (en) 2019-10-17 2023-12-07 Ginkgo Bioworks Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
US20210261625A1 (en) * 2020-01-29 2021-08-26 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
EP4135841A1 (en) 2020-04-15 2023-02-22 Voyager Therapeutics, Inc. Tau binding compounds
KR20230022175A (ko) 2020-05-13 2023-02-14 보이저 테라퓨틱스, 인크. Aav 캡시드의 향성 방향변경
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
US20230227802A1 (en) 2020-07-27 2023-07-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
CN111825772B (zh) * 2020-07-30 2023-10-20 中国科学院精密测量科学与技术创新研究院 具有变异衣壳蛋白的腺相关病毒及其应用
CN116390773A (zh) 2020-08-06 2023-07-04 应用医学研究基金会 用于通过基因疗法治疗诸如阿尔茨海默病的Tau蛋白病的病毒颗粒
US20230295657A1 (en) 2020-08-12 2023-09-21 UCB Biopharma SRL Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
KR20230092880A (ko) * 2020-08-28 2023-06-26 조인트 스탁 컴퍼니 "바이오케드" AAV5-기반의 SARS-CoV-2 백신
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
EP4225777A2 (en) * 2020-10-07 2023-08-16 RegenxBio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2022074105A1 (en) 2020-10-09 2022-04-14 UCB Biopharma SRL Nucleic acid constructs, viral vectors and viral particles
WO2022093769A1 (en) * 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
JP2023547992A (ja) 2020-10-28 2023-11-15 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 二重グリカン結合ａａｖ２．５ベクターのための方法および組成物
IL302608A (en) 2020-11-03 2023-07-01 Pfizer Methods for purification of aav vectors by anion exchange chromatography
WO2022101363A1 (en) 2020-11-11 2022-05-19 European Molecular Biology Laboratory Modified viral particles for gene therapy
US20240052322A1 (en) 2020-12-15 2024-02-15 Pfizer Inc. HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins
MX2023007527A (es) 2020-12-21 2023-07-10 Pfizer Metodos y sistemas para transfeccion celular mejorada.
AU2021404944A1 (en) 2020-12-23 2023-07-06 Pfizer Inc. Methods for purification of aav vectors by affinity chromatography
JP2024505739A (ja) 2021-02-10 2024-02-07 レジェンクスバイオ インコーポレーテッド 組換えヒトイズロン酸－２－スルファターゼ（ｉｄｓ）によるムコ多糖症ｉｉの治療
GB202101958D0 (en) 2021-02-12 2021-03-31 Ucl Business Ltd Gene therapy for dopamine transporter deficiency syndrome
CN112961219B (zh) * 2021-02-24 2022-08-12 中国农业科学院生物技术研究所 重组腺相关病毒、其突变体及其构建方法和应用
CN115044614B (zh) * 2021-03-09 2023-10-20 上海目镜生物医药科技有限公司 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用
US20240307553A1 (en) 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
US20250188126A1 (en) 2021-04-26 2025-06-12 Alexion Pharma International Operations Limited Adeno-associated viral vector capsids with improved tissue tropism
BR112023023249A2 (pt) * 2021-05-07 2024-01-23 Oyster Point Pharma Inc Fator neurotrófico de codificação de vírion aav e usos do mesmo
CA3218631A1 (en) 2021-05-12 2022-11-17 Alberto Auricchio Vector system
CA3224755A1 (en) 2021-06-22 2022-12-29 Pfizer Inc. Production of adeno-associated virus vector in insect cells
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
EP4419665A1 (en) 2021-10-20 2024-08-28 University of Rochester Rejuvenation treatment of age-related white matter loss
US20240417434A1 (en) 2021-10-28 2024-12-19 UCB Biopharma SRL Nucleic acid constructs, viral vectors and viral particles
US20250001012A1 (en) 2021-11-02 2025-01-02 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CA3236365A1 (en) 2021-11-02 2023-05-11 University Of Rochester Tcf7l2 mediated remyelination in the brain
TW202325850A (zh) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
KR20240116840A (ko) * 2021-12-28 2024-07-30 청두 오리젠 바이오테크놀로지 씨오., 엘티디. 변형된 aav 캡시드 단백질 및 이의 용도
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
TW202342526A (zh) 2022-02-21 2023-11-01 大陸商上海瑞宏迪醫藥有限公司 Ｖｅｇｆ結合分子及其醫藥用途
US12037616B2 (en) 2022-03-01 2024-07-16 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (ANGPTL3) related conditions
CN119562762A (zh) 2022-04-04 2025-03-04 加利福尼亚大学董事会 遗传互补组合物和方法
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
AR129843A1 (es) * 2022-07-06 2024-10-02 Voyager Therapeutics Inc Variantes de la cápside de aav y usos de estas
WO2024015877A2 (en) * 2022-07-12 2024-01-18 University Of Florida Research Foundation, Incorporated Novel aav3b capsid variants with enhanced hepatocyte tropism
KR20240014846A (ko) * 2022-07-26 2024-02-02 숙명여자대학교산학협력단 Aav2-anks1a 재조합 바이러스를 포함하는 알츠하이머 질병 치료용 조성물
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
CN116063404B (zh) * 2022-09-27 2025-08-29 广州译码基因科技有限公司 可提高AAV包装能力的衣壳蛋白MutD及其应用
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration
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
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
IL321778A (en) * 2023-01-05 2025-08-01 Emugen Therapeutics Llc Chimeric aav and uses thereof
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024189094A1 (en) 2023-03-14 2024-09-19 UCB Biopharma SRL Gene therapy
GB202304767D0 (en) * 2023-03-30 2023-05-17 Ucl Business Ltd Synthetic AAV Capsid
WO2024238853A1 (en) * 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2025022290A1 (en) 2023-07-21 2025-01-30 Crispr Therapeutics Ag Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene
TW202528335A (zh) * 2023-08-31 2025-07-16 美商戴諾治療公司 衣殼多肽及其使用方法(一)
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025108407A2 (en) 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
CN118440161B (zh) * 2024-01-16 2025-02-25 沈阳兴齐眼药股份有限公司 提高aav病毒视网膜穿透能力的衣壳蛋白突变体及其应用
WO2025186726A1 (en) 2024-03-05 2025-09-12 Crispr Therapeutics Ag Modulating expression of agt (angiotensinogen) gene
WO2025228976A1 (en) 2024-04-29 2025-11-06 UCB Biopharma SRL Gene therapy
GB202407038D0 (en) 2024-05-17 2024-07-03 Axovia Therapeutics Ltd Novel Gene Therapy
WO2025250457A1 (en) 2024-05-28 2025-12-04 University Of Rochester Enhanced brain transduction by gene therapeutics

Family Cites Families (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5639872A (en) 1993-07-27 1997-06-17 Hybridon, Inc. Human VEGF-specific oligonucleotides
AU1686997A (en) 1995-12-08 1997-06-27 Hybridon, Inc. Modified vegf antisense oligonucleotides for treatment of skin disorders
US20030215422A1 (en) 1996-09-11 2003-11-20 John A. Chiorini 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
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US20030144221A1 (en) 2001-07-17 2003-07-31 Isis Pharmaceuticals Inc. Antisense modulation of BCL2-associated X protein expression
AU2002360355B2 (en) 2001-11-09 2005-07-07 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
ES2258601T3 (es) 2001-11-13 2006-09-01 The Trustees Of The University Of Pennsylvania Un metodo para la identificacion de las secuencias desconocidas del virus adeno-asociado (vaa) y un kit para el metodo.
EP2573170B1 (en) 2001-12-17 2017-12-20 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor
EP2359869B1 (en) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
US20050106558A1 (en) 2001-12-21 2005-05-19 Luca Perabo Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
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
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
SI2657248T1 (sl) 2003-06-19 2017-07-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in njihova uporaba
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
EP3910063A1 (en) * 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP2899278A1 (en) 2004-03-12 2015-07-29 Alnylam Pharmaceuticals Inc. iRNA agents targeting VEGF
WO2006066066A2 (en) 2004-12-15 2006-06-22 University Of North Carolina At Chapel Hill Chimeric vectors
EP4234687A2 (en) * 2005-04-07 2023-08-30 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
US7543116B2 (en) 2006-01-30 2009-06-02 International Business Machines Corporation Data processing system, cache system and method for handling a flush operation in a data processing system having multiple coherency domains
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
US20090317417A1 (en) 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
ATE553206T1 (de) 2006-08-24 2012-04-15 Virovek Inc Expression von genen mit überlappenden offenen leserastern in insektenzellen, verfahren und zusammensetzungen dafür
US7872118B2 (en) 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
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
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
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
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
ES2634118T3 (es) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts 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
WO2011011572A1 (en) 2009-07-22 2011-01-27 Aware, Inc. Improved packet detector
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
CN102071206A (zh) * 2010-10-22 2011-05-25 中山大学 一种腺相关病毒衣壳蛋白基因、相应的蛋白及其应用
ES2638342T3 (es) * 2011-04-22 2017-10-19 The Regents Of The University Of California Viriones de virus adenoasociado con cápside variante y métodos para su uso
EP3147295B2 (en) 2011-08-24 2023-11-22 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
HUE054087T2 (hu) * 2012-04-18 2021-09-28 Childrens Hospital Philadelphia Készítmények és eljárások nagy hatékonyságú géntranszferre AAV kapszidvariánsok alkalmazásával
EP3470523A1 (en) 2012-05-09 2019-04-17 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
US10006049B2 (en) 2013-05-16 2018-06-26 University Of Florida Research Foundation, Incorporated Hairpin mRNA elements and methods for the regulation of protein translation
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11458211B2 (en) 2016-07-12 2022-10-04 The University Of Manchester Gene therapy

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020022476A (ja) * 2014-02-17 2020-02-13 キングス カレッジ ロンドンＫｉｎｇｓ Ｃｏｌｌｅｇｅ Ｌｏｎｄｏｎ アデノ随伴ウイルスベクター
US11802293B2 (en) 2014-02-17 2023-10-31 King's College London Adeno-associated virus vector

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