JP2023507174A5 - - Google Patents

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Publication number
JP2023507174A5
JP2023507174A5 JP2022537154A JP2022537154A JP2023507174A5 JP 2023507174 A5 JP2023507174 A5 JP 2023507174A5 JP 2022537154 A JP2022537154 A JP 2022537154A JP 2022537154 A JP2022537154 A JP 2022537154A JP 2023507174 A5 JP2023507174 A5 JP 2023507174A5
Authority
JP
Japan
Prior art keywords
dmd
target site
cells
intron
composition according
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
JP2022537154A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023507174A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/065437 external-priority patent/WO2021127057A1/en
Publication of JP2023507174A publication Critical patent/JP2023507174A/ja
Publication of JP2023507174A5 publication Critical patent/JP2023507174A5/ja
Pending legal-status Critical Current

Links

JP2022537154A 2019-12-16 2020-12-16 Ｄｍｄ変異の修正のための方法及び組成物 Pending JP2023507174A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962948665P 2019-12-16 2019-12-16
US62/948,665 2019-12-16
PCT/US2020/065437 WO2021127057A1 (en) 2019-12-16 2020-12-16 Methods and compositions for correction of dmd mutations

Publications (2)

Publication Number Publication Date
JP2023507174A JP2023507174A (ja) 2023-02-21
JP2023507174A5 true JP2023507174A5 (https=) 2023-12-26

Family

ID=76478088

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022537154A Pending JP2023507174A (ja) 2019-12-16 2020-12-16 Ｄｍｄ変異の修正のための方法及び組成物

Country Status (4)

Country Link
US (1) US20230272428A1 (https=)
EP (1) EP4076539A4 (https=)
JP (1) JP2023507174A (https=)
WO (1) WO2021127057A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2021345112A1 (en) * 2020-09-15 2023-04-27 Research Institute At Nationwide Children's Hospital Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
WO2025260086A1 (en) * 2024-06-14 2025-12-18 President And Fellows Of Harvard College In vivo site-specific base editing

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2684892A1 (en) * 2012-07-13 2014-01-15 Association Française contre les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
CN106714845A (zh) * 2014-08-11 2017-05-24 得克萨斯州大学系统董事会 通过crispr/cas9介导的基因编辑预防肌营养不良
US20170266320A1 (en) * 2014-12-01 2017-09-21 President And Fellows Of Harvard College RNA-Guided Systems for In Vivo Gene Editing
EP3288594B1 (en) * 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
AU2016344609B2 (en) * 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
AU2019216321A1 (en) * 2018-01-31 2020-07-30 The Board Of Regents Of The University Of Texas System Compositions and methods for correcting dystrophin mutations in human cardiomyocytes
WO2020214609A1 (en) * 2019-04-14 2020-10-22 Duke University Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
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

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