JP2023541444A5 - - Google Patents

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Publication number
JP2023541444A5
JP2023541444A5 JP2023516690A JP2023516690A JP2023541444A5 JP 2023541444 A5 JP2023541444 A5 JP 2023541444A5 JP 2023516690 A JP2023516690 A JP 2023516690A JP 2023516690 A JP2023516690 A JP 2023516690A JP 2023541444 A5 JP2023541444 A5 JP 2023541444A5
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JP
Japan
Prior art keywords
promoter
nucleotide sequence
nucleic acid
sequence shown
seq
Prior art date
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
JP2023516690A
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English (en)
Japanese (ja)
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JP2023541444A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/050461 external-priority patent/WO2022060841A2/en
Publication of JP2023541444A publication Critical patent/JP2023541444A/ja
Publication of JP2023541444A5 publication Critical patent/JP2023541444A5/ja
Pending legal-status Critical Current

Links

JP2023516690A 2020-09-15 2021-09-15 筋ジストロフィー患者における多様なdmd変異の補正のためのaav媒介性の相同性非依存的標的化組み込み遺伝子編集 Pending JP2023541444A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063078428P 2020-09-15 2020-09-15
US63/078,428 2020-09-15
US202163180232P 2021-04-27 2021-04-27
US63/180,232 2021-04-27
PCT/US2021/050461 WO2022060841A2 (en) 2020-09-15 2021-09-15 Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy

Publications (2)

Publication Number Publication Date
JP2023541444A JP2023541444A (ja) 2023-10-02
JP2023541444A5 true JP2023541444A5 (https=) 2024-09-20

Family

ID=78086092

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023516690A Pending JP2023541444A (ja) 2020-09-15 2021-09-15 筋ジストロフィー患者における多様なdmd変異の補正のためのaav媒介性の相同性非依存的標的化組み込み遺伝子編集

Country Status (6)

Country Link
US (1) US20230357795A1 (https=)
EP (1) EP4214317A2 (https=)
JP (1) JP2023541444A (https=)
AU (1) AU2021345112A1 (https=)
CA (1) CA3195233A1 (https=)
WO (1) WO2022060841A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023240157A2 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
CN115806989B (zh) * 2022-11-25 2023-08-08 昆明理工大学 针对DMD基因5号外显子突变的sgRNA及载体和应用

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WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
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CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
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WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
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US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
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
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
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DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
EP3004339B1 (en) 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
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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
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WO2016112242A1 (en) 2015-01-08 2016-07-14 President And Fellows Of Harvard College Split cas9 proteins
AU2016344609B2 (en) * 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
JP7075597B2 (ja) 2016-05-05 2022-05-26 デューク ユニバーシティ デュシェンヌ型筋ジストロフィーを治療するためのcrispr/cas関連の方法および組成物
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AR118668A1 (es) * 2019-04-14 2021-10-20 Univ Duke Composiciones para editar genoma a base de crispr / cas para restaurar la función de distrofina
US20230272428A1 (en) * 2019-12-16 2023-08-31 President And Fellows Of Harvard College Methods and compositions for correction of dmd mutations
US20230257723A1 (en) * 2020-04-27 2023-08-17 Duke University Crispr/cas9 therapies for correcting duchenne muscular dystrophy by targeted genomic integration

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