JP2023507174A - Ｄｍｄ変異の修正のための方法及び組成物 - Google Patents

Ｄｍｄ変異の修正のための方法及び組成物 Download PDF

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JP2023507174A
JP2023507174A JP2022537154A JP2022537154A JP2023507174A JP 2023507174 A JP2023507174 A JP 2023507174A JP 2022537154 A JP2022537154 A JP 2022537154A JP 2022537154 A JP2022537154 A JP 2022537154A JP 2023507174 A JP2023507174 A JP 2023507174A
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dmd
cell
grna
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intron
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エイミー ジェイ． ウェイジャーズ，
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Harvard University
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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

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

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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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521555A (ja) * 2013-06-05 2016-07-25 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
JP2018522530A (ja) * 2015-04-27 2018-08-16 ザ・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 ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系

Family Cites Families (7)

* 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
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
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521555A (ja) * 2013-06-05 2016-07-25 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
JP2018522530A (ja) * 2015-04-27 2018-08-16 ザ・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 ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系

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US20230272428A1 (en) 2023-08-31
EP4076539A1 (en) 2022-10-26
EP4076539A4 (en) 2024-06-05
WO2021127057A1 (en) 2021-06-24

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