JP2021521782A5 - - Google Patents

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Publication number
JP2021521782A5
JP2021521782A5 JP2020557155A JP2020557155A JP2021521782A5 JP 2021521782 A5 JP2021521782 A5 JP 2021521782A5 JP 2020557155 A JP2020557155 A JP 2020557155A JP 2020557155 A JP2020557155 A JP 2020557155A JP 2021521782 A5 JP2021521782 A5 JP 2021521782A5
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JP
Japan
Prior art keywords
helix
triple
raav
dystrophin
mutant protein
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Application number
JP2020557155A
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English (en)
Japanese (ja)
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JP2021521782A (ja
JP7575273B2 (ja
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Priority claimed from PCT/US2019/027675 external-priority patent/WO2019204303A2/en
Publication of JP2021521782A publication Critical patent/JP2021521782A/ja
Publication of JP2021521782A5 publication Critical patent/JP2021521782A5/ja
Application granted granted Critical
Publication of JP7575273B2 publication Critical patent/JP7575273B2/ja
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JP2020557155A 2018-04-16 2019-04-16 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法 Active JP7575273B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862658464P 2018-04-16 2018-04-16
US62/658,464 2018-04-16
PCT/US2019/027675 WO2019204303A2 (en) 2018-04-16 2019-04-16 Compositions and methods for treating duchenne muscular dystrophy

Publications (3)

Publication Number Publication Date
JP2021521782A JP2021521782A (ja) 2021-08-30
JP2021521782A5 true JP2021521782A5 (https=) 2022-04-19
JP7575273B2 JP7575273B2 (ja) 2024-10-29

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ID=68240632

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020557155A Active JP7575273B2 (ja) 2018-04-16 2019-04-16 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法

Country Status (6)

Country Link
US (2) US12460226B2 (https=)
EP (1) EP3780952A4 (https=)
JP (1) JP7575273B2 (https=)
CN (1) CN112601454B (https=)
CA (1) CA3099958A1 (https=)
WO (1) WO2019204303A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023510392A (ja) * 2020-01-16 2023-03-13 武田薬品工業株式会社 フェニルケトン尿症に対するアデノ随伴ウイルスベースの遺伝子治療
CN113999861B (zh) * 2021-09-08 2024-03-15 三峡大学 腺相关病毒衣壳蛋白保守区多克隆抗体的制备方法及应用

Family Cites Families (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69333886T2 (de) 1992-11-18 2006-07-27 Arch Development Corp., Chicago Adenovirus-gelenkter Gen-Transfer zum Herz-und glattem vaskulären Muskel
US20030148968A1 (en) 1995-02-28 2003-08-07 Hammond H. Kirk Techniques and compositions for treating cardiovascular disease by in vivo gene delivery
US6162796A (en) 1995-09-27 2000-12-19 The Rockefeller University Method for transferring genes to the heart using AAV vectors
GB9622174D0 (en) 1995-12-19 1996-12-18 Medical Res Council Gene expression
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US7078387B1 (en) 1998-12-28 2006-07-18 Arch Development Corp. Efficient and stable in vivo gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
EP1139751A4 (en) 1998-12-28 2003-03-19 Arch Dev Corp EFFICIENT AND STABLE -I (IN VIVO) GENE TRANSFER TO CARDIOMYCETS USING RECOMBINANT ADENO-ASSOCIATED VIRUS VECTORS
US20040192593A1 (en) 1999-07-26 2004-09-30 Baylor College Of Medicine Protease resistant ti-growth hormone releasing hormone
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
AU2001249315A1 (en) 2000-03-22 2001-10-03 Rheogene, Inc. Ecdysone receptor-based inducible gene expression system
ES2330615T3 (es) 2000-04-28 2009-12-14 Asklepios Biopharmaceutical, Inc. Secuencias de adn que codifican para minigenes de distrofina y metodos de uso de las mismas.
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
WO2002066614A2 (en) 2001-02-20 2002-08-29 Rheogene Holdings, Inc. Chimeric retinoid x receptors and their use in a novel ecdysone receptor-based inducible gene expression system
EP1373470B1 (en) 2001-02-20 2013-04-24 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
JP4955904B2 (ja) 2001-02-20 2012-06-20 イントレキソン コーポレーション 新規エクジソン受容体/無脊椎動物レチノイドx受容体−ベースの誘導性遺伝子発現システム
EP1534738B1 (en) 2001-02-20 2012-06-20 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
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
WO2005027995A2 (en) 2003-09-19 2005-03-31 The Trustees Of The University Of Pennsylvania Global myocardial perfusion catheter
WO2005030292A2 (en) 2003-09-26 2005-04-07 The Trustees Of The University Of Pennsylvania Methods, compositions and apparatus for delivering heterologous molecules to cells
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
US7771993B2 (en) 2004-01-23 2010-08-10 The Trustees Of The University Of Pennsylvania Microutrophin and uses thereof
US7935510B2 (en) 2004-04-30 2011-05-03 Intrexon Corporation Mutant receptors and their use in a nuclear receptor-based inducible gene expression system
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
US7774057B2 (en) 2005-09-06 2010-08-10 Cardiac Pacemakers, Inc. Method and apparatus for device controlled gene expression for cardiac protection
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
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
AU2014235096B2 (en) 2013-03-15 2020-03-19 The Trustees Of The University Of Pennsylvania Compositions and methods for treating MPSI
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
PL3097197T3 (pl) 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
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
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
SG11201808812RA (en) 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
CA2971303C (en) * 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
CA3037635A1 (en) * 2016-09-23 2018-03-29 Universite Laval Methods of modifying the dystrophin gene and restoring dystrophin expression and uses thereof

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