CN101711164B - 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 - Google Patents

可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 Download PDF

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CN101711164B
CN101711164B CN200880002602.3A CN200880002602A CN101711164B CN 101711164 B CN101711164 B CN 101711164B CN 200880002602 A CN200880002602 A CN 200880002602A CN 101711164 B CN101711164 B CN 101711164B
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dystrophin
nnos
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CN101711164A (zh
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段东升
赖毅
岳永平
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University of Missouri Columbia
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CN200880002602.3A 2007-01-18 2008-01-18 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 Active CN101711164B (zh)

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Application Number Priority Date Filing Date Title
US88112907P 2007-01-18 2007-01-18
US60/881,129 2007-01-18
US99932107P 2007-10-16 2007-10-16
US60/999,321 2007-10-16
PCT/US2008/000717 WO2008088895A2 (en) 2007-01-18 2008-01-18 Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma

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CN101711164A CN101711164A (zh) 2010-05-19
CN101711164B true CN101711164B (zh) 2014-06-04

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EP (1) EP2125006B1 (https=)
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Families Citing this family (84)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
US8236557B2 (en) * 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
US9169492B2 (en) 2010-02-05 2015-10-27 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
WO2013075008A1 (en) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
CN103405774A (zh) * 2012-10-24 2013-11-27 尹海芳 一种新型药物靶向运输辅助剂
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
US9624282B2 (en) * 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
ES2926774T3 (es) 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
WO2014143932A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
US10907176B2 (en) 2015-01-14 2021-02-02 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
JP6832280B2 (ja) * 2015-01-16 2021-02-24 ユニバーシティ オブ ワシントンUniversity of Washington 新規のマイクロジストロフィンおよび使用の関連する方法
HK1248554A1 (zh) 2015-02-20 2018-10-19 Board Of Regents, The University Of Texas System 作为疫苗和载体的减毒衣原体的方法和组合物
GB201507842D0 (en) * 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
EP3858993A1 (en) 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
KR102877920B1 (ko) 2015-11-16 2025-10-30 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
JP7406783B2 (ja) 2015-12-14 2023-12-28 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
ES2977730T3 (es) 2016-04-15 2024-08-29 Res Inst Nationwide Childrens Hospital Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular
LT3442600T (lt) 2016-04-15 2024-05-27 Research Institute At Nationwide Children's Hospital Su adeno virusu susijusio viruso vektoriaus iš β-sarkoglikano ir mikro- rnr-29 pristatymas ir raumenų distrofijos gydymas
WO2017223128A1 (en) * 2016-06-21 2017-12-28 The Curators Of The University Of Missouri Modified dystrophin proteins
CA2971303C (en) * 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
JP7165410B2 (ja) 2016-08-19 2022-11-04 ユニバーシティ オブ フロリダ リサーチ ファウンデーション,インコーポレイティド 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
KR20190088561A (ko) 2016-12-07 2019-07-26 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 인터류킨-1 수용체 길항제(IL-1RA) cDNA
WO2018162748A1 (en) 2017-03-10 2018-09-13 Genethon Treatment of glycogen storage disease iii
NZ756587A (en) 2017-03-15 2025-12-19 Univ North Carolina Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
EP3596112A2 (en) 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
MA50836A (fr) 2017-10-18 2020-08-26 Res Inst Nationwide Childrens Hospital Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire
KR102777452B1 (ko) 2017-11-22 2025-03-10 아지오스 파마슈티컬스 아이엔씨. N-(4-(4-(시클로프로필메틸)피페라진-1-카르보닐)페닐)퀴놀린-8-술폰아미드의결정 형태
AR114350A1 (es) 2018-01-31 2020-08-26 Res Institute At Nationwide Children’S Hospital Terapia genética para la distrofia muscular de cinturas del tipo 2c
EP3759218A4 (en) 2018-02-28 2021-12-08 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR ANTIBODY ELIMINATING VIRUS VECTORS
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
US12460226B2 (en) 2018-04-16 2025-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
CN112703016B (zh) 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
TW202016314A (zh) 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
MY208145A (en) * 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
EP3810782A2 (en) 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
AU2020229340A1 (en) 2019-02-26 2021-09-16 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
US12466864B2 (en) 2019-03-25 2025-11-11 Genethon Production of large-sized quasidystrophins using overlapping AAV vectors
EP4169535A1 (en) 2019-04-26 2023-04-26 The University of North Carolina at Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
AU2020284255A1 (en) 2019-05-30 2022-01-06 Solid Biosciences Inc. Recombinant herpesvirales vector
JP2022540819A (ja) * 2019-07-10 2022-09-20 ユニバーシティ オブ ワシントン 非ウイルス性免疫標的化
RS65421B1 (sr) 2019-08-21 2024-05-31 Res Inst Nationwide Childrens Hospital Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ,インコーポレイテッド ニーマン・ピック病c型の治療のためのアデノ随伴ウイルスベクター
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
WO2021089736A1 (en) 2019-11-06 2021-05-14 Association Institut De Myologie Combined therapy for muscular diseases
TWI908746B (zh) * 2019-11-28 2025-12-21 美商銳進科斯生物股份有限公司 微小肌縮蛋白基因療法之構築體及其用途
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
IL297753A (en) * 2020-04-29 2022-12-01 Bristol Myers Squibb Co Minimized dystrophins with spectrin fusion domains and uses thereof
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
WO2022093769A1 (en) 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
CN116490514A (zh) 2020-10-28 2023-07-25 北卡罗来纳-查佩尔山大学 用于双重聚糖结合aav2.5载体的方法和组合物
TW202242124A (zh) 2021-01-14 2022-11-01 美商史崔德生物公司 靶向t細胞之aav載體
EP4320139A1 (en) 2021-04-05 2024-02-14 Solid Biosciences Inc. Recombinant herpesvirales vector
WO2022221529A1 (en) 2021-04-16 2022-10-20 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
JP2024517143A (ja) * 2021-04-26 2024-04-19 リジェネックスバイオ インコーポレイテッド ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与
US20240390522A1 (en) * 2021-09-28 2024-11-28 University Of Florida Research Foundation, Incorporated Gene therapy for duchenne muscular dystrophy
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
CN115960946B (zh) * 2021-10-09 2026-02-13 中国科学院分子植物科学卓越创新中心 调节植物镁离子吸收和转运的方法
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CN119233831A (zh) * 2022-01-17 2024-12-31 迪纳柯公司 用于与肌营养不良蛋白有关的心肌病的基因疗法组合物和治疗
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
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
US20250288641A1 (en) * 2022-04-27 2025-09-18 The Curators Of The University Of Missouri Micro-dystrophin for heart protection
WO2024124019A2 (en) 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2025017168A1 (en) * 2023-07-19 2025-01-23 Genethon Novel optimized utrophin micro-genes

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1439051A (zh) * 2000-04-28 2003-08-27 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1366160B1 (en) * 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1439051A (zh) * 2000-04-28 2003-08-27 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
LAI Y ET AL.Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.《NATURE BIOTECHNOLOGY》.2005, *
LI SHENG ET AL.A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy.《HUMAN MOLECULAR GENETICS》.2006, *
RAFAEL J A ET AL.FORCED EXPRESSION OF DYSTROPHIN DELETION CONSTRCTS REVEALS STRUCTURE-FUNCTION CORRELATIONS.《THE JOURNAL OF CELL BIOLOGY》.1996, *
RAFAEL J A ET AL.PREVENTION OF DYSTROPHIC PATHOLOGY IN MDX MICE BY A TRUNCATED DYSTROPHIN ISOFORM.《HUMAN MOLECULAR GENETICS》.1994, *
SCOTT JEANNINE M ET AL.Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin.《NEUROMUSCULAR DISORDERS》.2002, *
WELLS KIM E ET AL.Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle.《NEUROMUSCULAR DISORDERS》.2003, *
Yi Lai et al.Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy.《The Journal of Clinical Investigation》.2009, *

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HK1143326A1 (en) 2010-12-31
EP2125006A4 (en) 2010-11-24
EP2125006A2 (en) 2009-12-02
EP2125006B1 (en) 2013-10-16
JP2010516252A (ja) 2010-05-20
CN101711164A (zh) 2010-05-19
US20080249052A1 (en) 2008-10-09
JP5575486B2 (ja) 2014-08-20

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