TWI908746B - 微小肌縮蛋白基因療法之構築體及其用途 - Google Patents

微小肌縮蛋白基因療法之構築體及其用途

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Publication number
TWI908746B
TWI908746B TW109141932A TW109141932A TWI908746B TW I908746 B TWI908746 B TW I908746B TW 109141932 A TW109141932 A TW 109141932A TW 109141932 A TW109141932 A TW 109141932A TW I908746 B TWI908746 B TW I908746B
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Taiwan
Prior art keywords
seq
aav
nucleic acid
acid sequence
protein
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TW109141932A
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English (en)
Chinese (zh)
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TW202134260A (zh
Inventor
春平 喬
迪文 麥道加
曄 劉
奧利斐爾 達諾斯
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美商銳進科斯生物股份有限公司
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TW109141932A 2019-11-28 2020-11-27 微小肌縮蛋白基因療法之構築體及其用途 TWI908746B (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962941719P 2019-11-28 2019-11-28
US62/941,719 2019-11-28
US202063024933P 2020-05-14 2020-05-14
US63/024,933 2020-05-14

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TW202134260A TW202134260A (zh) 2021-09-16
TWI908746B true TWI908746B (zh) 2025-12-21

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US (4) US20230270886A1 (https=)
EP (1) EP4065596A2 (https=)
JP (2) JP7822933B2 (https=)
KR (1) KR20220107222A (https=)
AU (1) AU2020392243A1 (https=)
BR (1) BR112022009895A2 (https=)
CA (1) CA3159516A1 (https=)
CL (1) CL2022001372A1 (https=)
MX (1) MX2022006427A (https=)
TW (1) TWI908746B (https=)
WO (1) WO2021108755A2 (https=)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL297753A (en) 2020-04-29 2022-12-01 Bristol Myers Squibb Co Minimized dystrophins with spectrin fusion domains and uses thereof
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
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
US20240269329A1 (en) * 2021-06-07 2024-08-15 University Of Florida Research Foundation, Incorporated Gene therapy for duchenne muscular dystrophy
AU2022297795A1 (en) * 2021-06-23 2024-01-04 AskBio Inc. Regulatory nucleic acid sequences
US20240409955A1 (en) * 2021-07-19 2024-12-12 New York University Auf1 combination therapies for treatment of muscle degenerative disease
WO2023018854A2 (en) * 2021-08-11 2023-02-16 Solid Biosciences Inc. Treatment of muscular dystrophy
US20250019721A1 (en) * 2021-08-11 2025-01-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
WO2023056367A1 (en) * 2021-09-30 2023-04-06 The Board Of Regents Of The Universityof Texas System Slc13a5 gene therapy vectors and uses thereof
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
US20240425572A1 (en) 2021-10-15 2024-12-26 Regenxbio Inc. Antibodies and methods of using thereof
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
WO2023141582A1 (en) 2022-01-21 2023-07-27 Regenxbio Inc. Engineered promoters
EP4230196A1 (en) 2022-02-21 2023-08-23 Som Innovation Biotech, S.A. Compounds for use in the treatment of dystrophinopathies
US20250213727A1 (en) 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
WO2023248251A1 (en) * 2022-06-24 2023-12-28 Indian Institute Of Technology Kanpur An optimized aav vector for gene therapy of muscular dystrophy
US20260055144A1 (en) 2022-08-24 2026-02-26 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
EP4601710A2 (en) 2022-10-11 2025-08-20 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2024091495A1 (en) * 2022-10-24 2024-05-02 Biogen Ma Inc. Compositions and methods for treating retinitis pigmentosa
WO2024138158A2 (en) 2022-12-23 2024-06-27 Ultragenyx Pharmaceutical Inc. Method of producing soluble microdystrophin protein and uses of the same
CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
AR133921A1 (es) 2023-09-25 2025-11-12 Sarepta Therapeutics Inc Distrofinas humanas adaptadas con mayor afinidad de unión a la actina para el tratamiento de las distrofias musculares
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav
WO2026080823A1 (en) 2024-10-10 2026-04-16 Research Institute At Nationwide Children's Hospital Methods for diagnosing and treating muscular dystrophy diseases

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016177911A1 (en) * 2015-05-07 2016-11-10 Royal Holloway & Bedford New College Production of large-sized microdystrophins in an aav-based vector configuration
CN107250364A (zh) * 2015-01-16 2017-10-13 华盛顿大学 新的微小肌养蛋白及相关使用方法

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
MX359371B (es) * 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US7183969B2 (en) 2004-12-22 2007-02-27 Raytheon Company System and technique for calibrating radar arrays
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
CN101711164B (zh) 2007-01-18 2014-06-04 密苏里-哥伦比亚大学 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
JP2011512156A (ja) 2008-02-19 2011-04-21 アムステルダム モレキュラー セラピューティクス ビー．ブイ． 昆虫細胞におけるパルボウイルスｒｅｐ及びｃａｐタンパク質の発現の最適化
EP2425000B1 (en) 2009-04-30 2019-02-20 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
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
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
MX388221B (es) 2013-04-20 2025-03-19 Res Institute At Nationwide Children´S Hospital ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2.
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
CN106232618A (zh) 2013-10-11 2016-12-14 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016100575A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
MX388739B (es) 2015-02-06 2025-03-20 Univ North Carolina Chapel Hill Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso.
JP6665466B2 (ja) 2015-09-26 2020-03-13 日亜化学工業株式会社 半導体発光素子及びその製造方法
WO2017070491A1 (en) 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
AU2016366560A1 (en) 2015-12-11 2018-06-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JP7676111B2 (ja) 2017-03-17 2025-05-14 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肉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
CN111902539B (zh) 2018-02-07 2025-05-16 吉尼松公司 杂合调控元件

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107250364A (zh) * 2015-01-16 2017-10-13 华盛顿大学 新的微小肌养蛋白及相关使用方法
WO2016177911A1 (en) * 2015-05-07 2016-11-10 Royal Holloway & Bedford New College Production of large-sized microdystrophins in an aav-based vector configuration

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