JP2023123765A5 - - Google Patents

Download PDF

Info

Publication number
JP2023123765A5
JP2023123765A5 JP2023108201A JP2023108201A JP2023123765A5 JP 2023123765 A5 JP2023123765 A5 JP 2023123765A5 JP 2023108201 A JP2023108201 A JP 2023108201A JP 2023108201 A JP2023108201 A JP 2023108201A JP 2023123765 A5 JP2023123765 A5 JP 2023123765A5
Authority
JP
Japan
Prior art keywords
aav
nucleic acid
particles
polypeptide
capsid
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2023108201A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023123765A (ja
Filing date
Publication date
Priority claimed from JP2017525107A external-priority patent/JP2017536116A/ja
Application filed filed Critical
Publication of JP2023123765A publication Critical patent/JP2023123765A/ja
Publication of JP2023123765A5 publication Critical patent/JP2023123765A5/ja
Priority to JP2025015920A priority Critical patent/JP2025072468A/ja
Pending legal-status Critical Current

Links

JP2023108201A 2014-11-21 2023-06-30 中枢神経系を標的化したaavベクター Pending JP2023123765A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025015920A JP2025072468A (ja) 2014-11-21 2025-02-03 中枢神経系を標的化したaavベクター

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201462082897P 2014-11-21 2014-11-21
US62/082,897 2014-11-21
US201562218857P 2015-09-15 2015-09-15
US62/218,857 2015-09-15
JP2017525107A JP2017536116A (ja) 2014-11-21 2015-11-20 中枢神経系を標的化したaavベクター
JP2021019477A JP2021097675A (ja) 2014-11-21 2021-02-10 中枢神経系を標的化したaavベクター

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2021019477A Division JP2021097675A (ja) 2014-11-21 2021-02-10 中枢神経系を標的化したaavベクター

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025015920A Division JP2025072468A (ja) 2014-11-21 2025-02-03 中枢神経系を標的化したaavベクター

Publications (2)

Publication Number Publication Date
JP2023123765A JP2023123765A (ja) 2023-09-05
JP2023123765A5 true JP2023123765A5 (enExample) 2023-11-24

Family

ID=56014589

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2017525107A Withdrawn JP2017536116A (ja) 2014-11-21 2015-11-20 中枢神経系を標的化したaavベクター
JP2021019477A Pending JP2021097675A (ja) 2014-11-21 2021-02-10 中枢神経系を標的化したaavベクター
JP2023108201A Pending JP2023123765A (ja) 2014-11-21 2023-06-30 中枢神経系を標的化したaavベクター
JP2025015920A Pending JP2025072468A (ja) 2014-11-21 2025-02-03 中枢神経系を標的化したaavベクター

Family Applications Before (2)

Application Number Title Priority Date Filing Date
JP2017525107A Withdrawn JP2017536116A (ja) 2014-11-21 2015-11-20 中枢神経系を標的化したaavベクター
JP2021019477A Pending JP2021097675A (ja) 2014-11-21 2021-02-10 中枢神経系を標的化したaavベクター

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025015920A Pending JP2025072468A (ja) 2014-11-21 2025-02-03 中枢神経系を標的化したaavベクター

Country Status (12)

Country Link
US (4) US10532110B2 (enExample)
EP (2) EP4344741A3 (enExample)
JP (4) JP2017536116A (enExample)
KR (2) KR20170086537A (enExample)
CN (2) CN114480440A (enExample)
AU (2) AU2015349759B2 (enExample)
BR (1) BR112017010447A2 (enExample)
CA (1) CA2967393A1 (enExample)
IL (1) IL252072B (enExample)
MX (1) MX2017006652A (enExample)
RU (1) RU2727015C2 (enExample)
WO (1) WO2016081811A1 (enExample)

Families Citing this family (69)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN112410338A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
EP4344741A3 (en) 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US10876134B2 (en) 2014-12-16 2020-12-29 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
RU2726131C2 (ru) 2015-05-22 2020-07-09 Дмитрий Дмитриевич Генкин Внеклеточная днк в качестве терапевтической мишени при нейродегенерации
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR20220108216A (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
KR102629418B1 (ko) 2016-06-13 2024-01-25 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 cln1 유전자 및 발현 카세트 및 그의 용도
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
EP3500676A4 (en) * 2016-08-16 2020-07-01 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR TRANSFERRING TARGETED GENES
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
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
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
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
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.
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
US11491241B2 (en) 2017-11-07 2022-11-08 The University Of North Carolina At Chapel Hill Optimized AGA genes and expression cassettes and their use
MX2020006473A (es) * 2017-12-20 2020-09-22 Ovid Therapeutics Inc Uso de hm4di en el tratamiento de trastornos convulsivos.
US11905522B2 (en) 2018-01-16 2024-02-20 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
CN108676097B (zh) * 2018-05-24 2020-01-14 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
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
SG11202103102WA (en) 2018-09-26 2021-04-29 California Inst Of Techn Adeno-associated virus compositions for targeted gene therapy
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
EP3861010A1 (en) * 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
US12270057B2 (en) 2018-11-05 2025-04-08 The University Of North Carolina At Chapel Hill Optimized FIG4 genes and expression cassettes and their use
JP7590968B2 (ja) 2018-12-05 2024-11-27 アビオナ・セラピュ―ティクス・インコーポレイテッド 遺伝子送達のための組み換えアデノ随伴ウイルスベクター
WO2020132385A1 (en) 2018-12-21 2020-06-25 The University Of North Carolina At Chapel Hill Optimized galc genes and expression cassettes and their use
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
MX2021013266A (es) * 2019-04-29 2021-11-17 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen.
EA202193214A1 (ru) 2019-05-22 2022-03-14 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Гены ube3a и кассеты экспрессии и их использование
US20220241394A1 (en) * 2019-07-12 2022-08-04 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
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
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
JP2023524061A (ja) * 2020-05-01 2023-06-08 ザ・ブロード・インスティテュート・インコーポレイテッド 操作された中枢神経系用組成物
WO2021225921A1 (en) * 2020-05-04 2021-11-11 The University Of North Carolina At Chapel Hill Modified adeno-associated virus vectors that evade neutralizing antibodies and uses thereof
MX2022013819A (es) * 2020-05-05 2023-01-30 Univ North Carolina Chapel Hill Capsides de virus adeno-asociado 5 modificado y usos del mismo.
AU2021273447B2 (en) 2020-05-13 2026-03-05 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
CN115925817A (zh) * 2020-05-22 2023-04-07 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
US20230265453A1 (en) 2020-07-08 2023-08-24 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
ES3057565T3 (en) 2020-07-23 2026-03-03 Univ North Carolina Chapel Hill Optimized slc13a5 genes and expression cassettes and their use
MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
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
IL311861A (en) 2021-11-02 2024-06-01 Voyager Therapeutics Inc Aav capsid variants and uses thereof
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

Family Cites Families (59)

* 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
EP0322417A1 (en) 1986-09-08 1989-07-05 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
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
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
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
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
AU762220B2 (en) 1998-05-28 2003-06-19 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The AAV5 vector and uses thereof
CA2347129A1 (en) 1998-09-22 2000-03-30 University Of Florida Methods for large-scale production of recombinant aav vectors
EP1127150B1 (en) 1998-11-05 2007-05-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
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
WO2001036603A2 (en) 1999-11-17 2001-05-25 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
CA2410901C (en) 2000-06-01 2012-03-20 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
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
ES2526341T3 (es) * 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
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.
US20070026076A1 (en) 2003-02-24 2007-02-01 Tzyy-Choou Wu 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
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
US7807814B2 (en) 2003-12-23 2010-10-05 The Trustees Of The University Of Pennsylvania Compositions and methods for combined therapy of disease
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
US20070059288A1 (en) 2005-03-31 2007-03-15 Dinsmore Jonathan H Treatment for heart disease
EP3085389A1 (en) 2005-04-07 2016-10-26 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US7867484B2 (en) * 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
ES2785223T3 (es) * 2006-06-21 2020-10-06 Uniqure Ip Bv Células de insecto para la producción de vectores de AAV
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
WO2009137006A2 (en) * 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP3567106A1 (en) 2010-04-23 2019-11-13 University of Massachusetts Cns targeting aav vectors and methods of use thereof
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
CN107828820B (zh) * 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
KR101830651B1 (ko) 2011-01-04 2018-02-21 엘지전자 주식회사 정보 표시 장치 및 그 방법
PL2900686T3 (pl) * 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
EP4344741A3 (en) 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system

Similar Documents

Publication Publication Date Title
JP2022530656A5 (enExample)
JP2022530544A5 (enExample)
JP2023076606A5 (enExample)
JP2017536116A5 (enExample)
JP6093358B2 (ja) アデノ随伴ウイルスベクターの産生細胞
JP2022033245A5 (enExample)
CN111349148A (zh) 一种腺相关病毒载体及其用途
JP2024514956A (ja) 組織標的化された改変aavカプシドおよびその使用方法
CN104937100A (zh) Aav变体
KR20180091863A (ko) 임상적 사용에 적합한 무혈청 현탁 세포 배양 시스템에서 재조합 아데노-관련 바이러스(aav) 벡터를 생성하기 위한 확장가능한 방법
JP2024051082A5 (enExample)
CN102947453A (zh) 用于病毒载体纯化的可扩缩生产平台和用于基因治疗中的如此纯化的病毒载体
CN115851837B (zh) 一种提高杆状病毒系统生产腺相关病毒的方法及应用
CN111876432B (zh) 一组肝靶向新型腺相关病毒的获得及其应用
EP3917566A1 (en) Methods for using transcription-dependent directed evolution of aav capsids
JP2023524577A5 (enExample)
JP7586547B2 (ja) 昆虫細胞内で重複オープンリーディングフレームを含む遺伝子を発現するための発現カセット及びその使用
CN106916793A (zh) 一种重组腺相关病毒的制备方法及重组杆状病毒
AU2021456513B2 (en) Expression cassette of gene comprising overlapping open reading frames and application thereof in insect cell
CN118420723A (zh) 提高aav病毒视网膜穿透能力的衣壳蛋白突变体及其应用
WO2024067153A1 (zh) 一种用于在昆虫细胞中产生rAAV的核酸、VP1衣壳蛋白突变体及应用
WO2023034990A1 (en) Aav capsid compositions and methods for delivery
CN108588097A (zh) 改造后的hbv基因组和相关组合物及其应用
WO2009125902A1 (en) Adeno associated virus serotype five vector for targeted gene delivery
CN116970650B (zh) 一种包膜蛋白组合、含其的靶向病毒载体及制备方法