JP7064214B2 - 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 - Google Patents

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 Download PDF

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JP7064214B2
JP7064214B2 JP2018515949A JP2018515949A JP7064214B2 JP 7064214 B2 JP7064214 B2 JP 7064214B2 JP 2018515949 A JP2018515949 A JP 2018515949A JP 2018515949 A JP2018515949 A JP 2018515949A JP 7064214 B2 JP7064214 B2 JP 7064214B2
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amino acid
aav
positions
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capsid protein
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JP2018528253A5 (enExample
JP2018528253A (ja
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アソカン，アラヴィンド
アグバンジェ‐マッケンナ，メイヴィス
ツェ，ロン・ピン・ヴィクター
ガーダ，ブリトニー
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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
ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/34—Identification of a linear epitope shorter than 20 amino acid residues or of a conformational epitope defined by amino acid residues
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    • C07K2317/00—Immunoglobulins specific features
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

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  • Neurology (AREA)
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  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2018515949A 2015-09-28 2016-09-28 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 Expired - Fee Related JP7064214B2 (ja)

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JP2022011956A JP7390693B2 (ja) 2015-09-28 2022-01-28 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
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US201562234016P 2015-09-28 2015-09-28
US62/234,016 2015-09-28
PCT/US2016/054143 WO2017058892A2 (en) 2015-09-28 2016-09-28 Methods and compositions for antibody-evading virus vectors

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Families Citing this family (98)

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Publication number Priority date Publication date Assignee Title
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
US11124546B2 (en) * 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
CA3033125A1 (en) * 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
KR20200067195A (ko) * 2017-10-18 2020-06-11 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항체 치료제
CA3092107A1 (en) * 2018-02-28 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
IL277270B2 (en) * 2018-03-16 2025-05-01 Res Inst Nationwide Childrens Hospital Increasing tissue-specific gene delivery through capsid modification
WO2019191716A1 (en) * 2018-03-29 2019-10-03 University Of Florida Research Foundation, Incorporated Aav6 variants
EP3781187A4 (en) * 2018-03-29 2021-12-22 Asklepios Biopharmaceutical, Inc. TROPICAL RECOMBINANT AAV6 LIVER VECTORS TO Bypass NEUTRALIZATION
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
BR112020020266A2 (pt) * 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
WO2019213668A1 (en) * 2018-05-04 2019-11-07 Oregon Health & Science University Human anti-aav2 capsid polyclonal antibody epitopes
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
JP7142815B2 (ja) * 2018-06-21 2022-09-28 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
AU2019354793A1 (en) 2018-10-05 2021-05-13 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding AAV production proteins
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
CA3125770A1 (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
CN113727992A (zh) * 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
AU2020299026A1 (en) * 2019-07-04 2022-01-20 Children's Medical Research Institute Methods and AAV vectors for in vivo transduction
JP7371954B2 (ja) * 2019-07-12 2023-10-31 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
CN110295151A (zh) * 2019-07-17 2019-10-01 博鑫仪器(天津)有限公司 一类腺依赖病毒病毒重组质粒、重组病毒及构建方法
EP4010465A1 (en) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4010357A2 (en) 2019-08-09 2022-06-15 University of Florida Research Foundation, Incorporated Aav capsid variants for targeting human glioblastoma cells
RU2751592C2 (ru) * 2019-08-22 2021-07-15 Общество С Ограниченной Ответственностью "Анабион" Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
EP4022070A1 (en) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
WO2021108467A1 (en) * 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target human hepatocytes and evade the humoral immune response
JP2021097617A (ja) * 2019-12-20 2021-07-01 学校法人自治医科大学 オルニチントランスカルバミラーゼ欠損症の治療用アデノ随伴ウイルスビリオン
TWI910162B (zh) * 2020-05-05 2026-01-01 美國公爵大學 跨物種相容腺相關病毒組合物及其使用方法
US12630842B2 (en) 2020-05-13 2026-05-19 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
JP2023542614A (ja) 2020-08-19 2023-10-11 サレプタ セラピューティクス， インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
CN111925438B (zh) * 2020-08-28 2021-03-09 和元生物技术（上海）股份有限公司 能够与aav1-13结合的抗体
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4328313A4 (en) * 2021-04-21 2025-06-25 Jichi Medical University Adeno-associated virus virion for treating ornithine transcarbamylase deficiency
WO2022229703A2 (en) * 2021-04-30 2022-11-03 Takeda Pharmaceutical Company, Ltd. New aav8 based immune escaping variants
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CA3238867A1 (en) 2021-11-29 2023-06-01 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
TW202342759A (zh) 2022-02-04 2023-11-01 美商史崔德生物公司 重組腺相關病毒載體及其使用方法
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
US20250161486A1 (en) * 2022-02-22 2025-05-22 The University Of North Carolina At Chapel Hill Chimeric neurotropic aav capsids
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN115850415B (zh) * 2022-09-14 2024-05-28 青岛农业大学 一种PbCaM7基因、蛋白及其应用
KR102855674B1 (ko) * 2022-10-06 2025-09-08 주식회사 글루진테라퓨틱스 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
JP2026509271A (ja) 2023-03-10 2026-03-17 ダイノ セラピューティクス，インコーポレーテッド カプシドポリペプチドおよびその使用方法
CN121039150A (zh) * 2023-03-13 2025-11-28 新加坡科技研究局 工程改造的腺相关病毒
CN121712809A (zh) 2023-07-03 2026-03-20 Ucb生物制药有限责任公司 工程化的aav衣壳蛋白
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
KR20260082361A (ko) 2023-08-31 2026-06-04 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
KR102844372B1 (ko) * 2023-12-06 2025-08-11 주식회사 글루진테라퓨틱스 뇌 성상교세포 특이적 유전자 전달이 가능한 아데노부속바이러스 변이체와 이의 응용
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
WO2025254596A1 (en) * 2024-06-06 2025-12-11 Agency For Science, Technology And Research Modified adeno-associated virus 1 (aav1)
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026122963A1 (en) 2024-12-06 2026-06-11 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN120842371B (zh) * 2025-09-25 2025-11-21 北京溯本源和生物科技有限公司 一种可广谱识别aav多种抗原的单克隆抗体5f11及应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050287122A1 (en) 2001-01-05 2005-12-29 Children's Hospital Inc. AAV vectors and methods
WO2015121501A1 (en) 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector

Family Cites Families (192)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1453547A (en) 1922-05-18 1923-05-01 Doyle Barrel-holding device
US1777296A (en) 1928-08-22 1930-10-07 Superior Locker Company Indicating device for locker doors
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
AU7966987A (en) 1986-09-08 1988-04-07 Applied Biotechnology, Inc. Empty viral capsid vaccines
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
EP0442926A1 (en) 1988-11-10 1991-08-28 Imperial Cancer Research Technology Limited Polypeptides
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
IL122206A0 (en) 1995-06-07 1998-04-05 Univ North Carolina Adeno-associated virus vectors for gene expression
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
EP0883344B2 (en) 1995-12-15 2010-06-09 VIRxSYS Corporation Therapeutic molecules generated by trans-splicing
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The 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
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
US6562958B1 (en) 1998-06-09 2003-05-13 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to Acinetobacter baumannii for diagnostics and therapeutics
US8299321B2 (en) 1998-06-16 2012-10-30 Monsanto Technology Llc Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20100293663A2 (en) 1998-06-16 2010-11-18 Thomas La Rosa Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
DE19830522A1 (de) 1998-07-08 2000-01-13 Pritt Produktionsgesellschaft Vorrichtung zum Übertragen eines in Form eines Filmes auf ein Trägerband aufgebrachten Stoffes
AU774706B2 (en) 1998-09-22 2004-07-08 Johns Hopkins University, The Methods for large-scale production of recombinant AAV vectors
GB9822763D0 (en) 1998-10-20 1998-12-16 Univ Sheffield Immunoglobin variant
DE69936104T2 (de) 1998-11-05 2008-01-17 The Trustees Of The University Of Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
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
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6822071B1 (en) 1998-11-12 2004-11-23 The Regents Of The University Of California Polypeptides from Chlamydia pneumoniae and their use in the diagnosis, prevention and treatment of disease
EP1887081A2 (en) 1999-02-25 2008-02-13 Ceres Incorporated DNA Sequences
EP1033405A3 (en) 1999-02-25 2001-08-01 Ceres Incorporated Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
US20110093981A9 (en) 1999-05-06 2011-04-21 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20080229439A1 (en) 1999-05-06 2008-09-18 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20040031072A1 (en) 1999-05-06 2004-02-12 La Rosa Thomas J. Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement
US20110214206A1 (en) 1999-05-06 2011-09-01 La Rosa Thomas J Nucleic acid molecules and other molecules associated with plants
US20130326723A1 (en) 1999-05-06 2013-12-05 Thomas J. La Rosa Soy nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20090087878A9 (en) 1999-05-06 2009-04-02 La Rosa Thomas J Nucleic acid molecules associated with plants
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US20140130203A1 (en) 2000-04-19 2014-05-08 Thomas J. La Rosa Rice nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20110131679A2 (en) 2000-04-19 2011-06-02 Thomas La Rosa Rice Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
JP2004520803A (ja) 2000-04-21 2004-07-15 コリクサ コーポレイション 尋常性挫瘡の治療および診断のための組成物および方法
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
JP2002130156A (ja) 2000-10-20 2002-05-09 Anest Iwata Corp 多段式流体圧縮部を備えたスクロール流体機械
US7214786B2 (en) 2000-12-14 2007-05-08 Kovalic David K Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
AU2002345250A1 (en) 2001-06-22 2003-01-08 Syngenta Participations Ag Plant disease resistance genes
WO2003008540A2 (en) 2001-06-22 2003-01-30 Syngenta Participations Ag Abiotic stress responsive polynucleotides and polypeptides
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
AU2001289843A1 (en) 2001-08-28 2002-02-13 Bayer Cropscience Ag Polypeptides for identifying herbicidally active compounds
WO2003033515A1 (en) 2001-10-15 2003-04-24 Corixa Corporation Compositions and methods for the therapy and diagnosis of acne vulgaris
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
CN103589692B (zh) 2001-11-13 2018-07-24 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
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
EP1463805B1 (en) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
US7071172B2 (en) 2002-04-30 2006-07-04 The University Of North Carolina At Chapel Hill Secretion signal vectors
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
JP2005185101A (ja) 2002-05-30 2005-07-14 National Institute Of Agrobiological Sciences 植物の全長ｃＤＮＡおよびその利用
DK1575517T3 (da) 2002-12-24 2012-05-14 Rinat Neuroscience Corp Anti-ngf-antistoffer og fremgangsmåder til anvendelse af samme
CA2528012C (en) 2003-06-02 2015-11-24 University Of Massachusetts Methods and compositions for controlling efficacy of rna silencing
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
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
US20060107345A1 (en) 2003-09-30 2006-05-18 Nickolai Alexandrov Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
KR101637908B1 (ko) 2004-04-07 2016-07-11 리나트 뉴로사이언스 코프. 신경성장인자 길항제의 투여에 의한 골암 통증의 치료방법
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
JP4346526B2 (ja) 2004-08-31 2009-10-21 株式会社東芝 半導体集積回路装置
US7366250B2 (en) 2004-09-09 2008-04-29 Agere Systems Inc. Method and apparatus for improved efficiency in an extended multiple antenna communication system
CA2579519A1 (en) 2004-09-09 2006-03-16 The General Hospital Corporation Modulating phosphatase activity in cardiac cells
EP1828390B1 (en) * 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
US20080213759A1 (en) 2005-01-05 2008-09-04 Shionogi & Co., Ltd. Novel Angiogenesis Inhibitor
US20140199313A1 (en) 2005-03-02 2014-07-17 Metanomics Gmbh Process for the Production of Fine Chemicals
WO2006092449A2 (en) 2005-03-02 2006-09-08 Metanomics Gmbh Process for the production of fine chemicals
AU2006242371A1 (en) 2005-04-29 2006-11-09 The University Of North Carolina At Chapel Hill Methods and compositions for regulated expression of nucleic acid at post-transcriptional level
EP1882392A4 (en) 2005-05-10 2009-07-01 Monsanto Technology Llc GENES AND THEIR USES FOR PLANT IMPROVEMENTS
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
US7867484B2 (en) * 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
JP2009525757A (ja) 2006-02-10 2009-07-16 ザ ユニバーシティ オブ シンシナティ 心機能のレギュレータとしてのホスファターゼインヒビタープロテイン−１
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
JP2009535339A (ja) * 2006-04-28 2009-10-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
WO2008088895A2 (en) 2007-01-18 2008-07-24 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
US9611302B2 (en) * 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
DK2623605T3 (en) * 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
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
BRPI0817005A2 (pt) 2007-09-18 2019-09-24 Basf Plant Science Gmbh método para produzir uma célula de planta transgênica, uma planta ou uma parte da mesma com rendimento aumentado, molécula de ácido nucleico, construção de ácido nucleico, vetor, célula hospedeira, processo para produzir um polipeptídeo, polipeptídeo, anticorpo, núcleo de célula de planta transgênica, célula de planta, tecido de planta, material de propagação, material colhido, planta ou parte da mesma, semente, processo para identificar um composto, método para produzir uma composição agrícola, composição, e, uso de uma molécula de ácido nucleico
EP2250268A4 (en) 2008-02-20 2011-08-24 Ceres Inc IMPROVED NITROGEN USE OF EFFICIENCY PROPERTIES IN PLANT NUCLEOTIDE SEQUENCES AND CORRESPONDING POLYPEPTIDES
WO2009108274A2 (en) 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
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
EP2396343B1 (en) * 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
SI3988660T1 (sl) 2009-05-02 2025-09-30 Genzyme Corporation Genska terapija za nevrodegenerativne motnje
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
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
US10738326B2 (en) 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
CA2826273C (en) * 2011-02-10 2021-11-02 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
ES2685611T3 (es) 2011-02-14 2018-10-10 The Children's Hospital Of Philadelphia Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo
AU2012298157B9 (en) 2011-08-23 2019-05-02 Evogene Ltd. Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics
FI124176B (en) 2011-12-01 2014-04-15 Helsingin Yliopisto Polypeptide, polynucleotide, expression cassette, vector, host cell, plant and uses
JP6342886B2 (ja) 2012-04-18 2018-06-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
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
US10294281B2 (en) * 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP2492347A1 (en) 2012-05-22 2012-08-29 Laboratorios Del. Dr. Esteve, S.A. Methods for the production of vectors
CN104704123A (zh) 2012-06-21 2015-06-10 肌肉学研究协会 基因治疗载体的广泛的基因递送
WO2014124282A1 (en) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
ES2926774T3 (es) 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
KR102298579B1 (ko) 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
NZ716102A (en) 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
HUE052676T2 (hu) 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
CN104592364B (zh) * 2013-10-30 2018-05-01 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
WO2015126972A1 (en) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
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
US10746742B2 (en) 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
US20170088852A1 (en) 2014-05-28 2017-03-30 Evogene Ltd. Isolated polynucleotides, polypeptides and methods of using same for increasing abiotic stress tolerance, biomass and yield of plants
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CA2953341C (en) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016054557A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Novel high efficiency library-identified aav vectors
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
AU2015349759B2 (en) 2014-11-21 2022-01-06 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
AU2016206624B2 (en) 2015-01-14 2020-08-06 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2016126857A1 (en) * 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
WO2016134338A1 (en) 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of human hematopoietic disorders
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
JP6836999B2 (ja) 2015-03-24 2021-03-03 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Ｔｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
EP3280451B8 (en) 2015-04-08 2019-09-25 The United States of America, as represented by the Secretary, Department of Health and Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
AU2016259976B2 (en) 2015-05-08 2021-10-14 Children's Medical Research Institute Promoters for expression of heterologous genes
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2016183297A1 (en) 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
CA2988471A1 (en) 2015-07-28 2017-02-02 University Of Massachusetts Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector
EP4134375A1 (en) 2015-07-30 2023-02-15 Massachusetts Eye & Ear Infirmary Ancestral adeno associated virus sequences and uses thereof
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
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP3384015A4 (en) 2015-12-01 2019-05-29 Spark Therapeutics, Inc. SCALABLE PROCESS FOR THE PREPARATION OF A RECOMBINANT ADENO ASSOCIATED VIRAL VECTOR IN A SERUM-FREE SUSPENSION CELL CULTURE SYSTEM SUITABLE FOR THE CLINICAL APPLICATION
WO2017096164A1 (en) 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
US11124546B2 (en) 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
CA3014637A1 (en) 2016-02-16 2017-08-24 Mark A. Kay Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
CN108697774A (zh) 2016-02-22 2018-10-23 北卡罗来纳大学教堂山分校 治疗mps i-相关失明的aav-idua载体
CN109661470A (zh) 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 新型aav8突变衣壳和含有其的组合物
WO2017192750A1 (en) 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
AU2017299779B2 (en) 2016-07-21 2021-12-02 Spark Therapeutics, Inc. Scalable high recovery methods for producing high yield recombinant adeno-associated viral (rAAV) vector and recombinant adeno-associated viral (rAAV) vectors produced thereby
JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
CA3033125A1 (en) 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
US11078238B2 (en) 2016-09-29 2021-08-03 University Of Florida Research Foundation, Incorporated AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US20230048025A1 (en) 2016-12-22 2023-02-16 Oregon Health & Science University Adeno associated viral vectors
EP3583220A4 (en) 2017-02-15 2021-07-07 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR GENERAL TRANSFER IN THE VASCULAR SYSTEM
RS65241B1 (sr) 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
WO2018170310A1 (en) 2017-03-15 2018-09-20 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
WO2018209154A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (app)-dependence of viruses
CA3068010A1 (en) 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Codon-optimized human npc1 genes for the treatment of niemann-pick type c1 deficiency and related conditions
CN111511375A (zh) 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
JP7389040B2 (ja) 2018-01-17 2023-11-29 メイラジーティーエックス・ユーケー・ザ・セカンド・リミテッド 遺伝子治療のための修飾ｒＡＡＶキャプシドタンパク質
EP3768695A4 (en) 2018-02-27 2022-04-06 The Trustees of the University of Pennsylvania NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
IL276859B2 (en) 2018-02-27 2025-12-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
CA3092107A1 (en) 2018-02-28 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
EP3762500A1 (en) 2018-03-06 2021-01-13 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
US11905524B2 (en) 2018-03-06 2024-02-20 University Of Florida Research Foundation, Incorporated AAV chimeras
IL277270B2 (en) 2018-03-16 2025-05-01 Res Inst Nationwide Childrens Hospital Increasing tissue-specific gene delivery through capsid modification
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050287122A1 (en) 2001-01-05 2005-12-29 Children's Hospital Inc. AAV vectors and methods
WO2015121501A1 (en) 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Journal of Virology，2012年，Vol.86, No.15，p.7739-7751
Molecular Therapy，2012年，Vol.20, No.2，p.329-338
Nature Communications，2014年，Vol.5, article No.3075，p.1-14
Selection of neutralizing antibody-resistant AAV8 variants with structure-guided site-specific saturated mutagenesis，Molecular Therapy，2011年，Vol.19 Suppl.1，S129

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