CN107532173A - 靶向中枢神经系统的aav载体 - Google Patents

靶向中枢神经系统的aav载体 Download PDF

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Publication number
CN107532173A
CN107532173A CN201580074023.XA CN201580074023A CN107532173A CN 107532173 A CN107532173 A CN 107532173A CN 201580074023 A CN201580074023 A CN 201580074023A CN 107532173 A CN107532173 A CN 107532173A
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Prior art keywords
aav
nucleic acid
particles
capsids
aav particles
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Pending
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CN201580074023.XA
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Chinese (zh)
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S.格雷
T.麦考恩
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University of North Carolina at Chapel Hill
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University of North Carolina at Chapel Hill
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Priority to CN202210236316.5A priority Critical patent/CN114480440A/zh
Publication of CN107532173A publication Critical patent/CN107532173A/zh
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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CN201580074023.XA 2014-11-21 2015-11-20 靶向中枢神经系统的aav载体 Pending CN107532173A (zh)

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Application Number Priority Date Filing Date Title
CN202210236316.5A CN114480440A (zh) 2014-11-21 2015-11-20 靶向中枢神经系统的aav载体

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US201462082897P 2014-11-21 2014-11-21
US62/082897 2014-11-21
US201562218857P 2015-09-15 2015-09-15
US62/218857 2015-09-15
PCT/US2015/061788 WO2016081811A1 (en) 2014-11-21 2015-11-20 Aav vectors targeted to the central nervous system

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US (4) US10532110B2 (https=)
EP (2) EP4344741A3 (https=)
JP (4) JP2017536116A (https=)
KR (2) KR20170086537A (https=)
CN (2) CN107532173A (https=)
AU (2) AU2015349759B2 (https=)
BR (1) BR112017010447A2 (https=)
CA (1) CA2967393A1 (https=)
IL (1) IL252072B (https=)
MX (1) MX2017006652A (https=)
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Cited By (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019223740A1 (zh) * 2018-05-24 2019-11-28 北京肽和生物科技有限公司 一种靶向肿瘤细胞的嵌合肽或嵌合蛋白及其应用
CN111574595A (zh) * 2020-05-22 2020-08-25 中国医学科学院血液病医院(中国医学科学院血液学研究所) 腺相关病毒突变体及其应用
CN112272672A (zh) * 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
CN112566923A (zh) * 2018-06-12 2021-03-26 北卡罗来纳大学教堂山分校 合成嗜肝性腺相关病毒衣壳及其用途
CN113166208A (zh) * 2018-10-02 2021-07-23 沃雅戈治疗公司 重新定向aav衣壳的嗜性
CN113423434A (zh) * 2018-12-05 2021-09-21 阿贝奥纳治疗有限公司 用于基因递送的重组腺相关病毒载体
CN113563430A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN115210248A (zh) * 2020-02-18 2022-10-18 北卡罗来纳大学查佩尔希尔分校 Aav衣壳-启动子相互作用和细胞选择性基因表达
CN115916986A (zh) * 2020-05-05 2023-04-04 北卡罗来纳大学查佩尔希尔分校 修饰的腺相关病毒5衣壳及其用途

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
WO2016081811A1 (en) 2014-11-21 2016-05-26 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
CN107249646B (zh) 2014-12-16 2021-06-29 内布拉斯加大学董事会 用于青少年巴滕病的基因疗法
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
WO2016190780A1 (en) 2015-05-22 2016-12-01 Dmitry Dmitrievich Genkin Extracellular dna as a therapeutic target in neurodegeneration
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
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
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
RU2764919C2 (ru) 2016-06-13 2022-01-24 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Оптимизированные гены и экспрессионные кассеты cln1, и их применение
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
US20190262373A1 (en) * 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
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
RU2020118342A (ru) 2017-11-07 2021-12-08 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Оптимизированные гены aga и экспрессионные кластеры и их применение
CN111867595A (zh) * 2017-12-20 2020-10-30 奥维德医疗公司 hM4Di在治疗癫痫发作紊乱中的用途
KR20200122320A (ko) 2018-01-16 2020-10-27 씨엘에스 테라퓨틱스 리미티드 데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
IL279131B2 (en) 2018-06-28 2025-06-01 Univ North Carolina Chapel Hill Optimized cln5 genes and expression cassettes and their use
IL280300B2 (en) 2018-08-10 2025-06-01 Univ North Carolina Chapel Hill Optimized cln7 genes and expression cassettes and their use
WO2020068990A1 (en) 2018-09-26 2020-04-02 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
BR112021006074A2 (pt) 2018-11-05 2021-07-20 The University Of North Carolina At Chapel Hill genes e cassetes de expressão fig4 otimizados e seu uso
EP3884057A4 (en) 2018-12-21 2022-09-07 The University of North Carolina at Chapel Hill OPTIMIZED GALC GENES AND EXPRESSION CASSETTE AND THEIR USE
PE20220014A1 (es) * 2019-04-29 2022-01-11 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen
EP3963073A4 (en) 2019-04-29 2023-01-25 The University of North Carolina at Chapel Hill OPTIMIZED SUMF1 GENES AND EXPRESSION CASSETTE AND THEIR USE
WO2020237130A1 (en) 2019-05-22 2020-11-26 The University Of North Carolina At Chapel Hill Ube3a genes and expression cassettes and their use
CA3147034A1 (en) * 2019-07-12 2021-01-21 Cls Therapeutics Limited Treatment of diseases associated with protein misfolding by nervous system expression of an enzyme which has a deoxyribonuclease (dnase) activity
WO2021041489A1 (en) * 2019-08-29 2021-03-04 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral vectors for crossing the human blood brain barrier
EP4025258A4 (en) * 2019-09-03 2023-09-06 University of Cincinnati METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
AU2021264061A1 (en) * 2020-05-01 2022-12-01 Massachusetts Institute Of Technology Engineered central nervous system compositions
US20230304033A1 (en) * 2020-05-04 2023-09-28 The University Of North Carolina At Chapel Hill Modified adeno-associated virus vectors that evade neutralizing antibodies and uses thereof
CN116096734A (zh) 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
EP4591708A3 (en) 2020-07-08 2026-01-14 Baylor College of Medicine Gene therapy for stxbp1 encephalopathy
US20230287458A1 (en) * 2020-07-14 2023-09-14 Abeona Therapeutics Inc. Recombinant adeno-associated viral vectors for multipartite gene delivery
US20230159949A1 (en) * 2020-07-22 2023-05-25 The Broad Institute, Inc. Engineered muscle targeting compositions
EP4185700B1 (en) 2020-07-23 2025-12-17 The University of North Carolina at Chapel Hill Optimized slc13a5 genes and expression cassettes and their use
TWI902867B (zh) * 2020-08-12 2025-11-01 比利時商Ｕｃｂ生物製藥公司 基因療法
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
MX2024004936A (es) 2021-11-02 2024-05-08 Voyager Therapeutics Inc Variantes de capsides de aav y sus usos.
US20250206783A1 (en) * 2022-03-21 2025-06-26 Capsigen Inc. Transcription-dependent directed evolution of aav capsids having enhanced tropism
AU2024294136A1 (en) * 2023-07-20 2026-02-05 Children's Medical Research Institute Modified cardiotropic aav capsid polypeptides and vectors
WO2025084951A1 (en) * 2023-10-16 2025-04-24 Joint Stock Company "Axelgen" Recombinant genetic construct, adeno-associated virus for the treatment of metachromatic leukodystrophy
WO2025147741A1 (en) * 2024-01-11 2025-07-17 Children's Medical Research Institute Aav capsids and vectors for transduction of cells

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007089632A2 (en) * 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
WO2011133890A8 (en) * 2010-04-23 2013-03-14 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2014052789A1 (en) * 2012-09-28 2014-04-03 The University Of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes

Family Cites Families (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4474893A (en) 1981-07-01 1984-10-02 The University of Texas System Cancer Center Recombinant monoclonal antibodies
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US4676980A (en) 1985-09-23 1987-06-30 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Target specific cross-linked heteroantibodies
AU7966987A (en) 1986-09-08 1988-04-07 Applied Biotechnology, Inc. Empty viral capsid vaccines
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
WO1990005142A1 (en) 1988-11-10 1990-05-17 Imperial Cancer Research Technology Ltd. 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
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
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
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
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
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
DE69636937T3 (de) 1995-12-15 2011-01-05 Virxsys Corp. Durch trans-spaltung erhaltene therapeutische molekule
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1082413B1 (en) 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav5 vector and uses thereof
AU774706B2 (en) 1998-09-22 2004-07-08 Johns Hopkins University, The Methods for large-scale production of recombinant AAV vectors
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
AU1775901A (en) 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
WO2001077350A2 (en) 2000-04-07 2001-10-18 Large Scale Biology Corporation Compositions and methods for inhibiting gene expression
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
US20030083299A1 (en) 2000-11-04 2003-05-01 Ferguson Ian A. Non-invasive delivery of polypeptides through the blood-brain barrier
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
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
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.
WO2005047501A1 (en) 2003-02-24 2005-05-26 Johns Hopkins University Molecular vaccines employing nucleic acid encoding anti-apoptotic proteins
ES2562490T3 (es) 2003-05-01 2016-03-04 Genzyme Corporation Terapia génica para trastornos neurometabólicos
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
CN1856576B (zh) * 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
EP1711172A4 (en) 2003-12-23 2008-07-16 Univ Pennsylvania COMPOSITIONS AND METHODS FOR COMBINATION THERAPY OF ILLNESSES
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
WO2006121532A2 (en) 2005-03-31 2006-11-16 Mytogen, Inc. Treatment for heart disease
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
KR101589259B1 (ko) * 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US8632764B2 (en) * 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
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
EP2634253B1 (en) * 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
KR101830651B1 (ko) 2011-01-04 2018-02-21 엘지전자 주식회사 정보 표시 장치 및 그 방법
WO2016081811A1 (en) * 2014-11-21 2016-05-26 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007089632A2 (en) * 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
WO2011133890A8 (en) * 2010-04-23 2013-03-14 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2014052789A1 (en) * 2012-09-28 2014-04-03 The University Of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
GRAY S J， ET AL: "Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood-Brain Barrier (BBB)", 《MOLECULAR THERAPY》 *
ZHIJIAN WU,ET AL: "Single Amino Acid Changes Can Influence Titer, Heparin Binding,and Tissue Tropism in Different Adeno-Associated Virus Serotypes", 《JOURNAL OF VIROLOGY》 *

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* Cited by examiner, † Cited by third party
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WO2019223740A1 (zh) * 2018-05-24 2019-11-28 北京肽和生物科技有限公司 一种靶向肿瘤细胞的嵌合肽或嵌合蛋白及其应用
US12410441B2 (en) 2018-06-12 2025-09-09 The University Of North Carolina At Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
CN112566923A (zh) * 2018-06-12 2021-03-26 北卡罗来纳大学教堂山分校 合成嗜肝性腺相关病毒衣壳及其用途
CN112566923B (zh) * 2018-06-12 2024-10-29 北卡罗来纳大学教堂山分校 合成嗜肝性腺相关病毒衣壳及其用途
CN113166208A (zh) * 2018-10-02 2021-07-23 沃雅戈治疗公司 重新定向aav衣壳的嗜性
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CN115210248A (zh) * 2020-02-18 2022-10-18 北卡罗来纳大学查佩尔希尔分校 Aav衣壳-启动子相互作用和细胞选择性基因表达
CN115916986A (zh) * 2020-05-05 2023-04-04 北卡罗来纳大学查佩尔希尔分校 修饰的腺相关病毒5衣壳及其用途
CN111574595B (zh) * 2020-05-22 2022-10-04 中国医学科学院血液病医院(中国医学科学院血液学研究所) 腺相关病毒突变体及其应用
CN116063403A (zh) * 2020-05-22 2023-05-05 中国医学科学院血液病医院(中国医学科学院血液学研究所) 腺相关病毒突变体及其应用
CN111574595A (zh) * 2020-05-22 2020-08-25 中国医学科学院血液病医院(中国医学科学院血液学研究所) 腺相关病毒突变体及其应用
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN113563430A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用

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