JP2023543029A5 - - Google Patents

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Publication number
JP2023543029A5
JP2023543029A5 JP2023519230A JP2023519230A JP2023543029A5 JP 2023543029 A5 JP2023543029 A5 JP 2023543029A5 JP 2023519230 A JP2023519230 A JP 2023519230A JP 2023519230 A JP2023519230 A JP 2023519230A JP 2023543029 A5 JP2023543029 A5 JP 2023543029A5
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JP
Japan
Prior art keywords
composition
approximately
use according
raav
item
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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
JP2023519230A
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English (en)
Japanese (ja)
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JP2023543029A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/052425 external-priority patent/WO2022067257A1/en
Publication of JP2023543029A publication Critical patent/JP2023543029A/ja
Publication of JP2023543029A5 publication Critical patent/JP2023543029A5/ja
Pending legal-status Critical Current

Links

JP2023519230A 2020-09-28 2021-09-28 筋ジストロフィーの治療のための製品及び方法 Pending JP2023543029A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063084259P 2020-09-28 2020-09-28
US63/084,259 2020-09-28
PCT/US2021/052425 WO2022067257A1 (en) 2020-09-28 2021-09-28 Products and methods for treating muscular dystrophy

Publications (2)

Publication Number Publication Date
JP2023543029A JP2023543029A (ja) 2023-10-12
JP2023543029A5 true JP2023543029A5 (https=) 2024-10-07

Family

ID=80846947

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023519230A Pending JP2023543029A (ja) 2020-09-28 2021-09-28 筋ジストロフィーの治療のための製品及び方法

Country Status (6)

Country Link
US (1) US20240261434A1 (https=)
EP (1) EP4217375A1 (https=)
JP (1) JP2023543029A (https=)
AU (1) AU2021349277A1 (https=)
CA (1) CA3194122A1 (https=)
WO (1) WO2022067257A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023228712A1 (en) 2022-03-04 2024-10-17 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising engineered small nuclear rna (snrna)
WO2025072530A2 (en) * 2023-09-26 2025-04-03 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating dmd

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP2001500497A (ja) 1996-09-06 2001-01-16 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
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
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
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.
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
CA2957661A1 (en) * 2014-08-09 2016-02-18 Kevin FLANIGAN Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法

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