KR20210031482A - 고-특이성의 게놈 편집을 달성하는 방법 - Google Patents

고-특이성의 게놈 편집을 달성하는 방법 Download PDF

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KR20210031482A
KR20210031482A KR1020217003832A KR20217003832A KR20210031482A KR 20210031482 A KR20210031482 A KR 20210031482A KR 1020217003832 A KR1020217003832 A KR 1020217003832A KR 20217003832 A KR20217003832 A KR 20217003832A KR 20210031482 A KR20210031482 A KR 20210031482A
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cas9
dna
cells
mrna
cas
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지우 왕
앤드류 엠. 참마스
알렉산더 와드
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알렐 바이오테크놀로지 앤 파마슈티칼스, 인크.
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KR1020217003832A 2018-07-13 2019-07-12 고-특이성의 게놈 편집을 달성하는 방법 Pending KR20210031482A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862697955P 2018-07-13 2018-07-13
US62/697,955 2018-07-13
PCT/US2019/041551 WO2020014577A1 (en) 2018-07-13 2019-07-12 Methods of achieving high specificity of genome editing

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KR20210031482A true KR20210031482A (ko) 2021-03-19

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US (1) US20220195403A1 (https=)
EP (1) EP3820503A4 (https=)
JP (2) JP7590952B2 (https=)
KR (1) KR20210031482A (https=)
CA (1) CA3106162A1 (https=)
TW (1) TW202023605A (https=)
WO (1) WO2020014577A1 (https=)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
EP4426828A1 (en) 2021-11-01 2024-09-11 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
AU2022420615A1 (en) 2021-12-22 2024-07-04 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
JP2025507990A (ja) * 2022-03-04 2025-03-21 エピジェニック・セラピューティクス・インコーポレイテッド ゲノム編集の組成物及び方法
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
CN115312122B (zh) * 2022-10-12 2022-12-16 之江实验室 一种CRISPR-Cas酶可突变位点推荐方法和装置
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
EP4677108A1 (en) 2024-04-22 2026-01-14 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013003475A1 (en) * 2011-06-27 2013-01-03 Cellscript, Inc. Inhibition of innate immune response
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
CN105492611A (zh) * 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CA2917348A1 (en) * 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
US9228207B2 (en) * 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
CA2969619A1 (en) * 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
EP3904366A1 (en) * 2014-12-16 2021-11-03 Novartis AG End capped nucleic acid molecules
CN104611368B (zh) * 2015-01-15 2018-05-25 中国科学院广州生物医药与健康研究院 重组后不产生移码突变的载体、在爪蛙基因组中进行基因定点敲入的方法及应用
EP3274454B1 (en) * 2015-03-25 2021-08-25 Editas Medicine, Inc. Crispr/cas-related methods, compositions and components
US10188750B1 (en) * 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
WO2017160662A1 (en) * 2016-03-12 2017-09-21 The Regents Of The University Of California Biodegradable vectors for efficient rna delivery
ES2962711T3 (es) * 2016-12-20 2024-03-20 Bristol Myers Squibb Co Métodos para aumentar la eficacia de la reparación dirigida por homología (HDR) en el genoma celular

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JP7590952B2 (ja) 2024-11-27
CA3106162A1 (en) 2020-01-16
JP2021530988A (ja) 2021-11-18
TW202023605A (zh) 2020-07-01
JP2024164090A (ja) 2024-11-26
EP3820503A1 (en) 2021-05-19
EP3820503A4 (en) 2022-07-13
US20220195403A1 (en) 2022-06-23
WO2020014577A1 (en) 2020-01-16

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