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
Authority
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.)
Pending
Application number
JP2021560108A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022526669A (ja
Filing date
Publication date
Application filed filed Critical
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
Pending legal-status Critical Current

Links

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 Pending JP2022526669A (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 (2)

Publication Number Publication Date
JP2022526669A JP2022526669A (ja) 2022-05-25
JP2022526669A5 true JP2022526669A5 (https=) 2023-04-24

Family

ID=72750921

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021560108A Pending JP2022526669A (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 (https=)
EP (1) EP3952884A4 (https=)
JP (1) JP2022526669A (https=)
WO (1) WO2020210776A1 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013163628A2 (en) 2012-04-27 2013-10-31 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
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods 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
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
US20240165271A1 (en) * 2021-03-26 2024-05-23 The Board Of Regents Of The University Of Texas System Nucleotide editing to reframe dmd transcripts by base editing and prime editing
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
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
WO2017035416A2 (en) * 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
KR102787119B1 (ko) * 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
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
EP3617311B1 (en) * 2017-03-30 2024-11-20 Kyoto University Method for inducing exon skipping by genome editing
AU2018251801B2 (en) * 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
CA3108281A1 (en) * 2018-08-03 2020-02-06 Beam Therapeutics Inc. Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence

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