HK1217730A1 - 衣殼修飾的 載體組合物及用於人肝癌基因治療中的應用方法 - Google Patents

衣殼修飾的 載體組合物及用於人肝癌基因治療中的應用方法

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Publication number
HK1217730A1
HK1217730A1 HK16105737.9A HK16105737A HK1217730A1 HK 1217730 A1 HK1217730 A1 HK 1217730A1 HK 16105737 A HK16105737 A HK 16105737A HK 1217730 A1 HK1217730 A1 HK 1217730A1
Authority
HK
Hong Kong
Prior art keywords
raav3
capsid
modified
methods
gene therapy
Prior art date
Application number
HK16105737.9A
Other languages
English (en)
Inventor
Arun Srivastava
Li Zhong
Sergei Zolotukhin
George V Aslanidi
Mavis Agbandje-Mckenna
Vliet Kim M Van
Original Assignee
Univ Florida
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
Priority claimed from US13/899,481 external-priority patent/US9920097B2/en
Application filed by Univ Florida filed Critical Univ Florida
Publication of HK1217730A1 publication Critical patent/HK1217730A1/zh

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HK16105737.9A 2013-05-21 2016-05-18 衣殼修飾的 載體組合物及用於人肝癌基因治療中的應用方法 HK1217730A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US13/899,481 US9920097B2 (en) 2007-04-09 2013-05-21 Capsid-modified rAAV vector compositions having improved transduction efficiencies, and methods of use
PCT/US2014/039015 WO2014193716A2 (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer

Publications (1)

Publication Number Publication Date
HK1217730A1 true HK1217730A1 (zh) 2017-01-20

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HK16105737.9A HK1217730A1 (zh) 2013-05-21 2016-05-18 衣殼修飾的 載體組合物及用於人肝癌基因治療中的應用方法

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EP (2) EP2999791B1 (zh)
JP (3) JP6628286B2 (zh)
KR (4) KR102234672B1 (zh)
CN (3) CN111763690A (zh)
AU (3) AU2014274457B2 (zh)
CA (1) CA2917018A1 (zh)
DK (1) DK2999791T3 (zh)
HK (1) HK1217730A1 (zh)
SG (2) SG10201709552XA (zh)
WO (1) WO2014193716A2 (zh)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5410017A (en) * 1993-05-21 1995-04-25 Rohm And Haas Company Continuous thermal polycondensation process for preparing polypeptide polymers
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
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
WO2017058892A2 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017070476A2 (en) * 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
BR112018011881A2 (pt) * 2015-12-14 2018-12-04 The University Of North Carolina At Chapel Hill proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
CA3033125A1 (en) * 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2018035503A1 (en) * 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated PROTEINS OF MODIFIED AAV CAPSIDES AND THEIR USES
PL3645551T3 (pl) * 2017-06-27 2024-07-01 Regeneron Pharmaceuticals, Inc. Zrekombinowane wektory wirusowe o zmodyfikowanym tropizmie i ich zastosowania do celowanego wprowadzania materiału genetycznego do komórek ludzkich
SG11202000765PA (en) * 2017-09-08 2020-03-30 Generation Bio Co Lipid nanoparticle formulations of non-viral, capsid-free dna vectors
WO2019143272A1 (en) * 2018-01-16 2019-07-25 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (dnase) activity
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF
US20210032660A1 (en) * 2018-03-29 2021-02-04 Asklepios Biopharmaceutical, Inc. Liver tropic recombinant aav6 vectors that evade neutralization
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
MX2020010466A (es) 2018-04-03 2021-01-08 Vectores de virus que evitan anticuerpos.
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
WO2019210267A2 (en) * 2018-04-27 2019-10-31 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
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
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
WO2020079256A1 (en) * 2018-10-19 2020-04-23 Genethon Modulation, monitoring and prediction of the immune response directed against aav gene therapy vectors
WO2020148458A1 (en) * 2019-01-18 2020-07-23 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
EP3736330A1 (en) * 2019-05-08 2020-11-11 European Molecular Biology Laboratory Modified adeno-associated virus (aav) particles for gene therapy
EP3999120A4 (en) * 2019-07-04 2024-02-21 Children's Medical Research Institute METHODS AND ADENO-ASSOCIATED VIRUS VECTORS FOR IN VIVO TRANSDUCTION
FI3997214T3 (fi) * 2019-07-11 2023-11-02 Centre Nat Rech Scient Kemiallisesti modifioitu adeno-associated virus
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
US20230340526A1 (en) * 2019-11-25 2023-10-26 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target hepatocytes and evade the humoral immune response
US20230049066A1 (en) * 2019-11-25 2023-02-16 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target human hepatocytes in the liver of humanized mice
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
AU2021267856A1 (en) * 2020-05-05 2022-12-01 The University Of North Carolina At Chapel Hill Modified adeno-associated virus 5 capsids and uses thereof
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
CN116648448A (zh) 2020-11-11 2023-08-25 欧洲分子生物学实验室 用于基因治疗的修饰病毒粒子
CN113647359A (zh) * 2021-08-15 2021-11-16 芜湖职业技术学院 注射衣霉素诱导急性肝损伤小鼠模型的构建方法
CN118201947A (zh) * 2021-10-28 2024-06-14 上海天泽云泰生物医药有限公司 腺相关病毒衣壳
WO2023201368A2 (en) * 2022-04-15 2023-10-19 Dyno Therapeutics, Inc. Capsid variants and methods of using the same

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US8999678B2 (en) * 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
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
ES2714007T3 (es) * 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
CA2762118A1 (en) * 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
EP4234571A3 (en) * 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
CN103060331B (zh) * 2012-12-05 2014-10-08 南京医科大学第一附属医院 介导膜稳定cd40l基因的衣壳蛋白突变的双链重组腺相关病毒制备方法与应用

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