JP2010516252A5 - - Google Patents

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Publication number
JP2010516252A5
JP2010516252A5 JP2009546436A JP2009546436A JP2010516252A5 JP 2010516252 A5 JP2010516252 A5 JP 2010516252A5 JP 2009546436 A JP2009546436 A JP 2009546436A JP 2009546436 A JP2009546436 A JP 2009546436A JP 2010516252 A5 JP2010516252 A5 JP 2010516252A5
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JP
Japan
Prior art keywords
nucleic acid
acid molecule
dystrophin protein
dystrophin
isolated nucleic
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JP2009546436A
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English (en)
Japanese (ja)
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JP2010516252A (ja
JP5575486B2 (ja
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Priority claimed from PCT/US2008/000717 external-priority patent/WO2008088895A2/en
Publication of JP2010516252A publication Critical patent/JP2010516252A/ja
Publication of JP2010516252A5 publication Critical patent/JP2010516252A5/ja
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Publication of JP5575486B2 publication Critical patent/JP5575486B2/ja
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JP2009546436A 2007-01-18 2008-01-18 筋鞘にnNOSを回復させる合成ミニ/ミクロジストロフィン遺伝子 Active JP5575486B2 (ja)

Applications Claiming Priority (5)

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

Publications (3)

Publication Number Publication Date
JP2010516252A JP2010516252A (ja) 2010-05-20
JP2010516252A5 true JP2010516252A5 (https=) 2011-03-10
JP5575486B2 JP5575486B2 (ja) 2014-08-20

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ID=39636602

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2009546436A Active JP5575486B2 (ja) 2007-01-18 2008-01-18 筋鞘にnNOSを回復させる合成ミニ/ミクロジストロフィン遺伝子

Country Status (5)

Country Link
US (1) US7892824B2 (https=)
EP (1) EP2125006B1 (https=)
JP (1) JP5575486B2 (https=)
CN (1) CN101711164B (https=)
WO (1) WO2008088895A2 (https=)

Families Citing this family (82)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method 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
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
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
CA2788682C (en) 2010-02-05 2019-03-05 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
WO2014081855A1 (en) * 2012-11-20 2014-05-30 Universite De Montreal 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
JP6396988B2 (ja) 2013-03-15 2018-09-26 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 二重グリカン結合性aavベクターのための方法および組成物
US9447433B2 (en) 2013-03-15 2016-09-20 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
AU2016206624B2 (en) 2015-01-14 2020-08-06 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
IL286316B2 (en) 2015-01-16 2023-03-01 Univ Washington New micro-dystrophins and related method of use
MX2017010698A (es) 2015-02-20 2018-04-30 Univ Texas Metodos y composiciones para chlamydia atenuada como vacuna y vector.
GB201507842D0 (en) * 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
EP3858993A1 (en) 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
CA3005633C (en) 2015-11-16 2023-11-21 Research Institute Of Nationwide Children's Hospital Materials and methods for treatment of titin-based myopathies and other titinopathies
EP4659816A3 (en) 2015-12-14 2025-12-31 The University of North Carolina at Chapel Hill MODIFIED CAPSID PROTEINS FOR IMPROVED PARVOVIRUS VECTOR DELIVERY
AU2017250791B2 (en) * 2016-04-15 2022-11-24 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
PT3442600T (pt) 2016-04-15 2024-05-09 Res Inst Nationwide Childrens Hospital Distribuição de vetor de vírus adeno-associado de bsarcoglicano e microrna-29 e o tratamento da distrofia muscular
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
PE20190401A1 (es) 2016-07-26 2019-03-13 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado
CN109843306A (zh) 2016-08-19 2019-06-04 卡琳缪恩股份有限公司 使用自身互补型重组腺相关病毒治疗病症的方法和组合物
KR20250121159A (ko) 2016-12-07 2025-08-11 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 인터류킨-1 수용체 길항제(IL-1RA) cDNA
US11541131B2 (en) 2017-03-10 2023-01-03 Genethon Treatment of glycogen storage disease III
CN110770346B (zh) 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
WO2019012336A2 (en) 2017-03-17 2019-01-17 Newcastle University ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
JP7213238B2 (ja) 2017-10-18 2023-01-26 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達
MX2020007876A (es) 2018-01-31 2020-12-03 Res Inst Nationwide Childrens Hospital Terapia genica para la distrofia muscular de cinturas tipo 2c.
BR112020016699A2 (pt) 2018-02-28 2020-12-15 The University Of North Carolina At Chapel Hill Métodos e composições para vetores de vírus de evasão de anticorpos
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
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, 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
CN119410636A (zh) 2018-04-27 2025-02-11 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
BR112020022722A8 (pt) 2018-05-09 2022-01-18 Biomarin Pharm Inc Genoma e seu uso, vetor, partícula, molécula de ácido nucleico isolada, ácido nucleico, composição, tratamento, método
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
CN120393053A (zh) * 2018-06-18 2025-08-01 全国儿童医院研究所 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送
EP3810782A2 (en) 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
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
AU2020218547A1 (en) 2019-02-08 2021-08-26 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
MX2021010356A (es) 2019-02-26 2022-03-04 Res Inst Nationwide Childrens Hospital SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR.
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
US12466864B2 (en) 2019-03-25 2025-11-11 Genethon Production of large-sized quasidystrophins using overlapping AAV vectors
EP3959227A1 (en) 2019-04-26 2022-03-02 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
EP4578503A3 (en) * 2019-07-10 2025-09-03 University of Washington Non-viral immuno-targeting
PT4017871T (pt) 2019-08-21 2024-04-22 Res Inst Nationwide Childrens Hospital Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
AU2020378619B2 (en) 2019-11-06 2026-03-05 Association Institut De Myologie Combined therapy for muscular diseases
MX2022006427A (es) * 2019-11-28 2022-09-07 Regenxbio Inc "construcciones para terapia génica con microdistrofina y uso de las mismas.
AU2021247172A1 (en) 2020-04-01 2022-09-15 University Of Florida Research Foundation, Incorporated Dual AAV-MY07A vectors with improved safety for the treatment of USH1B
AU2021265103A1 (en) 2020-04-29 2023-01-19 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
EP4200408A1 (en) 2020-08-19 2023-06-28 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
ES3001131T3 (es) 2020-10-28 2025-03-04 Univ North Carolina Chapel Hill Métodos y composiciones para la unión dual de glicanos al vector de AAV2.5
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
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
US20240191251A1 (en) 2021-04-05 2024-06-13 Solid Biosciences Inc. Recombinant herpesvirales vector
JP2024515626A (ja) 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル, インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドaavビリオン
KR20240004564A (ko) * 2021-04-26 2024-01-11 리젠엑스바이오 인크. 디스트로핀병증의 치료를 위한 마이크로디스트로핀 유전자 치료법 투여
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
JP2023059858A (ja) 2021-10-15 2023-04-27 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
CA3248878A1 (en) * 2022-01-17 2023-07-20 Dinaqor Ag GENE THERAPY COMPOSITION AND TREATMENT OF DYSTROPHIN-RELATED CARDIOMYOPATHY
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
WO2023150687A1 (en) 2022-02-04 2023-08-10 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors, and methods of use thereof
WO2023212193A1 (en) * 2022-04-27 2023-11-02 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
EP4694904A2 (en) 2023-04-13 2026-02-18 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

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE437944T1 (de) * 2000-04-28 2009-08-15 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung
ATE400656T1 (de) * 2000-10-06 2008-07-15 Univ Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen

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