JP2022526669A5 - - Google Patents

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Publication number
JP2022526669A5
JP2022526669A5 JP2021560108A JP2021560108A JP2022526669A5 JP 2022526669 A5 JP2022526669 A5 JP 2022526669A5 JP 2021560108 A JP2021560108 A JP 2021560108A JP 2021560108 A JP2021560108 A JP 2021560108A JP 2022526669 A5 JP2022526669 A5 JP 2022526669A5
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JP
Japan
Prior art keywords
cas
crispr
editing system
base editing
based base
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.)
Granted
Application number
JP2021560108A
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English (en)
Japanese (ja)
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JP2022526669A (ja
JP7881154B2 (ja
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Priority claimed from PCT/US2020/027867 external-priority patent/WO2020210776A1/en
Publication of JP2022526669A publication Critical patent/JP2022526669A/ja
Publication of JP2022526669A5 publication Critical patent/JP2022526669A5/ja
Application granted granted Critical
Publication of JP7881154B2 publication Critical patent/JP7881154B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2021560108A 2019-04-12 2020-04-12 ジストロフィン機能を修復するためのCRISPR/Casをベースにした塩基編集組成物 Active JP7881154B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962833454P 2019-04-12 2019-04-12
US62/833,454 2019-04-12
PCT/US2020/027867 WO2020210776A1 (en) 2019-04-12 2020-04-12 Crispr/cas-based base editing composition for restoring dystrophin function

Publications (3)

Publication Number Publication Date
JP2022526669A JP2022526669A (ja) 2022-05-25
JP2022526669A5 true JP2022526669A5 (enExample) 2023-04-24
JP7881154B2 JP7881154B2 (ja) 2026-06-29

Family

ID=72750921

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021560108A Active JP7881154B2 (ja) 2019-04-12 2020-04-12 ジストロフィン機能を修復するためのCRISPR/Casをベースにした塩基編集組成物

Country Status (4)

Country Link
US (1) US20220177879A1 (enExample)
EP (1) EP3952884A4 (enExample)
JP (1) JP7881154B2 (enExample)
WO (1) WO2020210776A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
KR20240132120A (ko) 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏(duchenne)肌肉失養症之大型突變熱點之aav載體介導之刪除
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
WO2022204476A1 (en) * 2021-03-26 2022-09-29 The Board Of Regents Of The University Of Texas System Nucleotide editing to reframe dmd transcripts by base editing and prime editing
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
CN119912582B (zh) * 2024-06-17 2025-11-04 中国农业大学 单碱基编辑器及其所用脱氨酶与应用

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
KR20240132120A (ko) * 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
WO2017070632A2 (en) * 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
US12214054B2 (en) * 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
WO2018017751A1 (en) * 2016-07-19 2018-01-25 Supereye, Inc. Systems and methods for predictive visual rendering
JP7490211B2 (ja) * 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
AU2017364106A1 (en) * 2016-11-28 2019-06-20 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by CRISPR/Cpfl-mediated gene editing
JOP20190166A1 (ar) * 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
CN110892069B (zh) * 2017-03-30 2024-02-09 国立大学法人京都大学 基于基因组编辑的外显子跳跃诱导方法
AU2018251801B2 (en) * 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
KR102894715B1 (ko) * 2018-08-03 2025-12-03 빔 테라퓨틱스, 인크. 핵산 표적 서열을 변형시키기 위한 다중-이펙터 핵염기 편집기 및 이를 이용하는 방법

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