MA44740A - Variantes de capsides de virus adéno-associé et leurs procédés d'utilisation - Google Patents

Variantes de capsides de virus adéno-associé et leurs procédés d'utilisation

Info

Publication number
MA44740A
MA44740A MA044740A MA44740A MA44740A MA 44740 A MA44740 A MA 44740A MA 044740 A MA044740 A MA 044740A MA 44740 A MA44740 A MA 44740A MA 44740 A MA44740 A MA 44740A
Authority
MA
Morocco
Prior art keywords
adeno
variants
methods
associated virus
capside
Prior art date
Application number
MA044740A
Other languages
English (en)
Inventor
David H Kirn
Melissa Kotterman
David Schaffer
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 MA44740A publication Critical patent/MA44740A/fr

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    • 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
    • A61K48/0016—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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • A61K39/12—Viral antigens
    • A61K39/235—Adenoviridae
    • 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
    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61K48/005—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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/10—Antioedematous agents; Diuretics
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14141—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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Dermatology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
MA044740A 2016-05-13 2017-05-12 Variantes de capsides de virus adéno-associé et leurs procédés d'utilisation MA44740A (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662336441P 2016-05-13 2016-05-13
US201662378106P 2016-08-22 2016-08-22
US201662384590P 2016-09-07 2016-09-07
US201762454612P 2017-02-03 2017-02-03

Publications (1)

Publication Number Publication Date
MA44740A true MA44740A (fr) 2019-02-27

Family

ID=60267403

Family Applications (1)

Application Number Title Priority Date Filing Date
MA044740A MA44740A (fr) 2016-05-13 2017-05-12 Variantes de capsides de virus adéno-associé et leurs procédés d'utilisation

Country Status (27)

Country Link
US (9) US11364308B2 (fr)
EP (3) EP4206216B1 (fr)
JP (1) JP6592208B2 (fr)
KR (1) KR102234930B1 (fr)
CN (3) CN116333057A (fr)
AU (1) AU2017261812B2 (fr)
BR (1) BR112018072849B1 (fr)
CA (1) CA3023592C (fr)
CL (1) CL2018003196A1 (fr)
CO (1) CO2018013255A2 (fr)
CR (1) CR20180589A (fr)
DK (2) DK3445773T5 (fr)
ES (1) ES2941502T3 (fr)
FI (2) FI4206216T3 (fr)
IL (1) IL262922B (fr)
MA (1) MA44740A (fr)
MX (2) MX2018013463A (fr)
NZ (1) NZ748395A (fr)
PE (1) PE20190129A1 (fr)
PH (1) PH12018502387A1 (fr)
PT (2) PT4206216T (fr)
RU (1) RU2725286C2 (fr)
SA (1) SA518400418B1 (fr)
SG (1) SG11201809684YA (fr)
UA (1) UA124343C2 (fr)
WO (1) WO2017197355A2 (fr)
ZA (2) ZA201807625B (fr)

Families Citing this family (92)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
DK4005603T3 (da) 2011-04-22 2024-11-25 Univ California Adeno-associerede virus-virioner med variant kapsid og fremgangsmåder til anvendelse heraf
CA2907799A1 (fr) 2013-05-31 2014-12-04 The Regents Of The University Of California Variants de virus adeno-associes et leurs methodes d'utilisation
ES3042252T3 (en) 2014-03-17 2025-11-19 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression in cone cells
CN114480502A (zh) 2015-03-02 2022-05-13 阿德夫拉姆生物技术股份有限公司 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
WO2016154344A1 (fr) 2015-03-24 2016-09-29 The Regents Of The University Of California Variants de virus adéno-associé et leurs procédés d'utilisation
JP7064214B2 (ja) 2015-09-28 2022-05-10 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 抗体回避性ウイルスベクターのための方法および組成物
CN116333057A (zh) * 2016-05-13 2023-06-27 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
CA3029833A1 (fr) 2016-07-29 2018-02-01 The Regents Of The University Of California Virions de virus adeno-associe a variant de capside et methodes d'utilisation associees
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
BR112019019015A2 (pt) * 2017-06-30 2020-04-14 Univ California vírions de vírus adeno-associado com capsídeos variantes e seus métodos de uso
TWI820034B (zh) 2017-07-31 2023-11-01 香港商映像生物有限公司 眼部疾病之細胞模式及用於眼部疾病的療法
WO2019040586A1 (fr) * 2017-08-25 2019-02-28 Ovid Therapeutics Inc. Vecteurs adéno-associés recombinants
EP3676373A4 (fr) 2017-08-28 2021-06-09 The Regents of The University of California Variants de capside de virus adéno-associé et leurs procédés d'utilisation
BR112020005436B1 (pt) * 2017-09-20 2022-08-02 4D Molecular Therapeutics Inc Proteína do capsídeo de variante do vírus adenoassociado, virion do aav recombinante (raav) infeccioso, composições, composições farmacêuticas e usos de virion de raav ou de composições farmacêuticas
US11723988B2 (en) * 2017-10-20 2023-08-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
FI3717636T3 (fi) * 2017-11-27 2023-06-01 4D Molecular Therapeutics Inc Adenoassosioidun viruksen kapsidivariantteja ja käyttö angiogeneesin estämisessä
JP2021516048A (ja) 2018-02-28 2021-07-01 ユニバーシティ オブ ピッツバーグ −オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション 遺伝子及びタンパク質の送達のためのａａｖに基づくモジュールシステム
WO2019178412A1 (fr) * 2018-03-16 2019-09-19 Research Institute At Nationwide Children's Hospital Augmentation de l'administration de gènes spécifiques d'un tissu par modification de capside
US12091435B2 (en) 2018-04-03 2024-09-17 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
JP2021523702A (ja) * 2018-05-04 2021-09-09 オレゴン ヘルス アンド サイエンス ユニバーシティ ヒト抗ａａｖ２カプシドポリクローナル抗体エピトープ
CN112839709A (zh) * 2018-06-06 2021-05-25 加利福尼亚大学董事会 治疗神经退行性疾病的神经干细胞组合物和方法
GB201809588D0 (en) * 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
US20210290727A1 (en) * 2018-08-01 2021-09-23 University Of Maryland, Baltimore MODULATION OF mTORCI ACTIVITY AND AUTOPHAGY VIA CIB2-RHEB INTERACTION
US12195745B2 (en) * 2018-08-21 2025-01-14 Massachusetts Eye And Ear Infirmary Compositions and methods for modulating transduction efficiency of adeno-associated viruses
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
WO2020077091A1 (fr) * 2018-10-10 2020-04-16 Wisconsin Alumni Research Foundation Vecteurs de thérapie génique kir 7.1 et procédés d'utilisation de ces derniers
US20220073905A1 (en) * 2018-12-28 2022-03-10 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods and materials for single cell transcriptome-based development of aav vectors and promoters
KR20210130158A (ko) 2019-01-31 2021-10-29 오레곤 헬스 앤드 사이언스 유니버시티 Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법
CN113728108A (zh) * 2019-02-15 2021-11-30 桑格摩生物治疗股份有限公司 用于生产重组aav的组合物和方法
JP7698583B2 (ja) 2019-03-21 2025-06-25 ギンコ バイオワークス インコーポレイテッド 組換えアデノ随伴ウイルスベクター
SG11202110607WA (en) * 2019-04-01 2021-10-28 Tenaya Therapeutics Inc Adeno-associated virus with engineered capsid
US20200360491A1 (en) * 2019-04-08 2020-11-19 The Children's Hospital Of Philadelphia Treatment of lysosomal storage disease in the eye through administration of aavs expressing tpp1
CA3136872A1 (fr) * 2019-04-23 2020-10-29 Inserm (Institut National De La Sante Et De La Recherche Medicale) Nouveaux variants du virus adeno-associe (aav) et leurs utilisations en therapie genique
AU2020263757B2 (en) 2019-04-24 2025-08-28 Takara Bio Inc. AAV mutant having brain-targeting property
TWI862583B (zh) 2019-04-26 2024-11-21 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼
EP3736330A1 (fr) * 2019-05-08 2020-11-11 European Molecular Biology Laboratory Particules modifiées du virus adéno-associé (aav) pour la thérapie génique
WO2021009805A1 (fr) * 2019-07-12 2021-01-21 株式会社遺伝子治療研究所 Virion de virus adéno-associé pour transfert de gènes vers le foie humain
EP3999119B1 (fr) * 2019-07-15 2024-09-11 Meiragtx UK II Limited Protéines de capside aav modifiées pour le traitement des maladies arthritiques
AU2020347276A1 (en) * 2019-09-12 2022-03-17 Massachusetts Institute Of Technology Engineered adeno-associated virus capsids
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for treatment of niemann-pick disease type c
US20230002788A1 (en) * 2019-10-21 2023-01-05 The Trustees Of The University Of Pennsylvania Aav3b variants with improved production yield and liver tropism
CN110724673B (zh) * 2019-10-31 2021-08-06 上海爱尔眼科医院有限公司 嗜外层视网膜的腺相关病毒病毒体及其应用
US12509705B2 (en) 2019-10-31 2025-12-30 Universität Bern AAV vector variants for ocular gene delivery
GB202005641D0 (en) * 2020-04-17 2020-06-03 Ucl Business Plc Gene therapy for bardet-biedl syndrome
US20230139443A1 (en) * 2020-02-28 2023-05-04 The Trustees Of The University Of Pennsylvania Treating autosomal dominant bestrophinopathies and methods for evaluating same
WO2021184009A1 (fr) * 2020-03-13 2021-09-16 Wisconsin Alumni Research Foundation Vecteurs de thérapie génique kir7.1 et leurs méthodes d'utilisation
EP4121443A4 (fr) * 2020-03-20 2024-05-15 Dignity Health Procédé d'ingénierie et d'isolement de virus adéno-associé
KR20230026986A (ko) 2020-04-27 2023-02-27 4디 몰레큘러 테라퓨틱스 아이엔씨. 코돈 최적화된 gla 유전자 및 이의 용도
CA3179597A1 (fr) * 2020-05-29 2021-12-02 John G. Flannery Virions de virus adeno-associes avec des capsides variants et leurs procedes d'utilisation
WO2022011390A1 (fr) * 2020-07-08 2022-01-13 Baylor College Of Medicine Thérapie génique pour encéphalopathie stxbp1
CN111849998A (zh) * 2020-07-29 2020-10-30 武汉纽福斯生物科技有限公司 编码人卵黄状黄斑病蛋白1的核酸分子及其应用
CN111733174B (zh) * 2020-08-07 2021-02-09 北京大学第三医院（北京大学第三临床医学院） 一种分离的核酸分子及其用途
CA3189878A1 (fr) 2020-08-19 2022-02-24 Colin O'BANION Vecteurs de type virus adeno-associe pour le traitement du syndrome de rett
KR102825852B1 (ko) * 2020-09-02 2025-06-26 4디 몰레큘러 테라퓨틱스 아이엔씨. 코돈 최적화된 rpgrorf15 유전자 및 이의 용도
WO2022087104A1 (fr) * 2020-10-21 2022-04-28 Chan Zuckerberg Biohub, Inc. Virions de virus adéno-associés et leurs procédés d'utilisation
AU2021378075B2 (en) 2020-11-11 2025-03-27 Borea Therapeutics S.R.L. Modified viral particles for gene therapy
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
US12385064B2 (en) 2021-03-26 2025-08-12 Adverum Biotechnologies, Inc. Intravitreal dosing for delivery of polynucleotides to retinal cones
EP4330411B1 (fr) 2021-04-27 2026-03-18 4D Molecular Therapeutics Inc. Compositions et méthodes pour le traitement d'une maladie oculaire associée à l'angiogenèse
CN117157309A (zh) * 2021-05-28 2023-12-01 上海瑞宏迪医药有限公司 衣壳变异的重组腺相关病毒及其应用
WO2023283962A1 (fr) * 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Capside aav modifiée pour thérapie génique et méthodes associées
JP2024529408A (ja) * 2021-07-23 2024-08-06 デューク ユニバーシティ アデノ随伴ウイルス組成物およびその使用方法
WO2023044306A1 (fr) * 2021-09-14 2023-03-23 California Institute Of Technology Variants capsidiques de vaa
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
CN114057840B (zh) * 2021-10-14 2022-09-20 和元生物技术（上海）股份有限公司 包含变异的aav9衣壳蛋白的重组腺相关病毒颗粒
WO2023106256A1 (fr) * 2021-12-06 2023-06-15 学校法人順天堂 Vecteur de virus adéno-associé modifié
CN120187407A (zh) * 2021-12-15 2025-06-20 健达九州(北京)生物科技有限公司 重组aav载体及其用途
CN120590489A (zh) * 2022-04-12 2025-09-05 合肥星眸生物科技有限公司 一种融合型腺相关病毒及其应用
WO2023201364A2 (fr) * 2022-04-15 2023-10-19 Dyno Therapeutics, Inc. Variants de capside et leurs procédés d'utilisation
EP4514984A1 (fr) * 2022-04-25 2025-03-05 Beijing Hanmoluojie Technology Co., Ltd. Cartographie de caractéristiques de surface moléculaire 3d de capsides d'aav
EP4555095A2 (fr) * 2022-07-14 2025-05-21 Fondation Asile Des Aveugles Thérapie génique pour rétinopathies associées à fam161a et autres ciliopathies
WO2024017387A1 (fr) * 2022-07-22 2024-01-25 Shanghai Vitalgen Biopharma Co., Ltd. Nouvelles capsides d'aav pour cibler le système nerveux et leurs utilisations
CN115968834A (zh) * 2023-01-20 2023-04-18 北京因诺惟康医药科技有限公司 一种rs1点突变小鼠模型的制备方法及其用途
CN118420721A (zh) * 2023-01-31 2024-08-02 上海朗昇生物科技有限公司 一种组织嗜性的腺相关病毒衣壳及其用途
TW202502803A (zh) 2023-03-10 2025-01-16 美商戴諾治療公司 衣殼多肽及其使用方法
WO2024211416A2 (fr) * 2023-04-07 2024-10-10 4D Molecular Therapeutics Inc. Variants d'aav pour le traitement de troubles de complément
WO2024220966A1 (fr) * 2023-04-21 2024-10-24 The Regents Of The University Of California Nouveaux composés de glycane pour la dégénérescence maculaire liée à l'âge
CN121219325A (zh) * 2023-05-23 2025-12-26 北京因诺惟康医药科技有限公司 一种腺相关病毒衣壳蛋白、包含其的腺相关病毒、载体及应用
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN120230798A (zh) * 2023-12-29 2025-07-01 上海朗昇生物科技有限公司 用于视网膜基因递送的重组腺相关病毒载体及其应用
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
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025214359A1 (fr) * 2024-04-09 2025-10-16 Hangzhou Jiayin Biotech Ltd. Nouveau variant de vaa
CN118222631B (zh) * 2024-05-24 2024-10-15 广州湾区生物基因科技有限公司 用于靶向治疗遗传性视网膜变性的病毒载体组合
CN119751599B (zh) * 2024-07-16 2025-08-19 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2026024048A1 (fr) * 2024-07-22 2026-01-29 한국생명공학연구원 Virus adéno-associé comprenant une capside variante et utilisation associée
CN118667036B (zh) * 2024-08-20 2025-01-10 上海玮美基因科技有限责任公司 一种融合型腺相关病毒衣壳蛋白、腺相关病毒及其应用

Family Cites Families (91)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0932694A2 (fr) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Vecteur de vaa4 et ses utilisations
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
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
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
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
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''
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US6498244B1 (en) * 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
WO2001029058A1 (fr) 1999-10-15 2001-04-26 University Of Massachusetts Genes de voies d'interference d'arn en tant qu'outils d'interference genetique ciblee
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
AU6486201A (en) 2000-05-23 2001-12-03 Univ Rochester Method of producing herpes simplex virus amplicons, resulting amplicons, and their use
CA2410947A1 (fr) 2000-05-30 2001-12-06 Johnson & Johnson Research Pty Limited Methodes permettant d'agir sur la suppression de gene par utilisation de facteurs renforcant l'arni
AU2001284160A1 (en) 2000-08-19 2002-03-04 Axordia Limited Modulation of stem cell differentiation
AU9307501A (en) 2000-09-22 2002-04-02 Virxsys Improved conditionally replicating vectors, methods for their production and use
WO2002029858A2 (fr) 2000-09-29 2002-04-11 Infineon Technologies North America Corp. Procede de gravure de tranchees profondes destine a reduire ou eliminer la formation de silicium noir
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
WO2002053703A2 (fr) 2001-01-05 2002-07-11 Children's Hospital, Inc. Vecteurs aav2 et procedes
WO2003042361A2 (fr) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production d'un virus adeno-associe dans des cellules d'insectes
CN103555677B (zh) 2001-11-13 2018-01-30 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
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
WO2003052051A2 (fr) 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Sequences du serotype 8 du virus associe a l'adenovirus (aav), vecteurs les contenant et utilisations correspondantes
EP1456383B1 (fr) 2001-12-21 2014-03-12 Medigene AG Bibliotheque de genes structuraux modifies ou de particules modifiees par des capsides utiles pour l'identification de clones viraux par tropisme cellulaire desire
WO2003088899A2 (fr) 2002-04-05 2003-10-30 The Children's Hospital Of Philadelphia Techniques de production de vecteurs de virus adeno-associes (aav) chimeriques, de composition de vecteurs aav chimeriques et techniques d'utilisation de ceux-ci
WO2003089612A2 (fr) 2002-04-17 2003-10-30 University Of Florida Research Foundation, Inc. Vecteurs raav ameliores utilises pour augmenter la transduction de cellules exprimant des recepteurs de lipoproteine a basse densite
EP1496944B1 (fr) 2002-05-01 2008-08-20 University of Florida Research Foundation, Inc. Systemes d'expression raav ameliores destines a une modification genetique de proteines de capside specifiques
US20060127358A1 (en) * 2002-05-01 2006-06-15 Nicholas Muzyczka Raav expression systems and methods for enhancing transduction of mammalian neural cells
US20040023390A1 (en) 2002-08-05 2004-02-05 Davidson Beverly L. SiRNA-mediated gene silencing with viral vectors
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
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 (fr) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Vecteur derivé d'un virus associé aux adenovirus pour la thérapie génique
US8071028B2 (en) 2003-06-12 2011-12-06 Abbott Diabetes Care Inc. Method and apparatus for providing power management in data communication systems
JP2007524386A (ja) 2003-06-19 2007-08-30 アビジェン， インコーポレイテッド 減少した免疫反応性を有するａａｖビリオン、およびその使用
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
DK3211085T3 (da) 2003-09-30 2021-06-21 Univ Pennsylvania Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf
DE602005020815D1 (de) 2004-09-24 2010-06-02 Intercell Ag Verändertes vp-1capsidprotein von parvovirus b19
US7892809B2 (en) 2004-12-15 2011-02-22 The University Of North Carolina At Chapel Hill Chimeric vectors
US8999678B2 (en) 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
WO2006119432A2 (fr) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, clonage, et caracterisation de nouveaux serotypes de virus adeno-associes (avv)
EP2311967B1 (fr) 2005-10-20 2017-09-20 UniQure IP B.V. Vecteurs viraux adéno-associés améliorés, produits en cellules d'insecte
WO2007089632A2 (fr) 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Vecteurs chimères de liaison à l'héparine et au sulfate d'héparane
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
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
EP2623605B1 (fr) 2007-04-09 2018-11-28 University of Florida Research Foundation, Inc. Compositions de vecteur RAAV comprenant des protéines de capside à tyrosine modifié et procédés d'utilisation
WO2008124015A1 (fr) 2007-04-09 2008-10-16 The Regents Of The University Of California Procédés de purification de virions de virus adéno-associés
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
EP2012122A1 (fr) 2007-07-06 2009-01-07 Medigene AG Protéines structurelles de parvovirus muté
US8299215B2 (en) 2007-07-14 2012-10-30 University Of Iowa Research Foundation 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
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker 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 (fr) 2008-09-19 2010-03-25 Charitē Universitätsmedizin Berlin Identification et caractérisation de vecteurs de thérapie génique viraux recombinants
EP2396343B1 (fr) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Vecteurs viraux modifiés et procédés de fabrication et d'utilisation de ceux-ci
WO2010138263A2 (fr) 2009-05-28 2010-12-02 University Of Massachusetts Nouveaux virus adéno-associés (aav) et leurs utilisations
US9133479B2 (en) 2009-06-03 2015-09-15 Cedars-Sinai Medical Center Effective vector platform for gene transfer and gene therapy
WO2011038187A1 (fr) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Diversification de virus adéno-associé (aav) commandé et bibliothèques préparées à partir de ce dernier
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
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
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
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
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
JP6042825B2 (ja) 2011-02-10 2016-12-14 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 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法
US10392632B2 (en) 2011-02-14 2019-08-27 The Children's Hospital Of Philadelphia AAV8 vector with enhanced functional activity and methods of use thereof
ES2724800T3 (es) 2011-02-17 2019-09-16 Univ Pennsylvania Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9
DK4005603T3 (da) 2011-04-22 2024-11-25 Univ California Adeno-associerede virus-virioner med variant kapsid og fremgangsmåder til anvendelse heraf
US9169299B2 (en) 2011-08-24 2015-10-27 The Board Of Trustees Of The Leleand Stanford Junior University AAV 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
WO2013123503A1 (fr) 2012-02-17 2013-08-22 The Children's Hospital Of Philadelphia Compositions de vecteurs de virus adéno-associés et méthodes de transfert de gènes dans des cellules, des organes et des tissus
RU2683497C2 (ru) 2012-04-18 2019-03-28 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav
EP2660325A3 (fr) 2012-05-02 2014-02-12 Christian Medical College Vecteurs de virus AAV et séquences de nucléotides et procédés correspondants
WO2013170078A1 (fr) 2012-05-09 2013-11-14 Oregon Health & Science University Plasmides et vecteurs viraux associés à un adénovirus
WO2013174760A1 (fr) 2012-05-25 2013-11-28 Deutsches Krebsforschungszentrum Vecteurs aav optimisés pour des taux de transduction élevés dans des cellules dendritiques
EP2692731A1 (fr) 2012-07-31 2014-02-05 Paul-Ehrlich-Institut Bundesamt für Sera und Impfstoffe Particules de vecteur AAV présentant des ligands à affinité élevée pour l'administration de gènes spécifique du type cellulaire
EP2900686B1 (fr) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Vecteurs vaa ciblés sur des oligodendrocytes
EP2940131B1 (fr) 2012-12-25 2019-02-20 Takara Bio Inc. Variant d'aav
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
EP2970946A4 (fr) 2013-03-13 2016-09-07 Philadelphia Children Hospital Vecteurs viraux adéno-associés et méthodes d'utilisation associées
CA2907799A1 (fr) 2013-05-31 2014-12-04 The Regents Of The University Of California Variants de virus adeno-associes et leurs methodes d'utilisation
US10308957B2 (en) 2014-03-04 2019-06-04 University Of Florida Research Foundation, Inc. rAAV vectors and methods for transduction of photoreceptors and RPE cells
WO2015164757A1 (fr) * 2014-04-25 2015-10-29 Oregon Health & Science University Procedes de cartographie d'epitopes d'anticorps de neutralisation virale
CN116333057A (zh) 2016-05-13 2023-06-27 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
CA3029833A1 (fr) * 2016-07-29 2018-02-01 The Regents Of The University Of California Virions de virus adeno-associe a variant de capside et methodes d'utilisation associees

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PE20190129A1 (es) 2019-01-17
AU2017261812B2 (en) 2019-04-18
IL262922B (en) 2019-09-26
RU2018142768A (ru) 2020-06-16
EP3445773B1 (fr) 2022-12-28
BR112018072849B1 (pt) 2020-12-22
NZ748395A (en) 2019-12-20
US11179477B2 (en) 2021-11-23
CA3023592A1 (fr) 2017-11-16
WO2017197355A2 (fr) 2017-11-16
US20230001018A1 (en) 2023-01-05
ES2941502T3 (es) 2023-05-23
US20210069349A1 (en) 2021-03-11
EP3445773A2 (fr) 2019-02-27
US20210177990A1 (en) 2021-06-17
CA3023592C (fr) 2020-12-08
DK3445773T3 (da) 2023-03-20
AU2017261812A1 (en) 2018-11-15

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