KR102604903B1 - 내인성 및 공급원 DNA의 원-샷 가이드 RNA(ogRNA) 표적화를 위한 시스템 및 방법 - Google Patents

내인성 및 공급원 DNA의 원-샷 가이드 RNA(ogRNA) 표적화를 위한 시스템 및 방법 Download PDF

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KR102604903B1
KR102604903B1 KR1020197019386A KR20197019386A KR102604903B1 KR 102604903 B1 KR102604903 B1 KR 102604903B1 KR 1020197019386 A KR1020197019386 A KR 1020197019386A KR 20197019386 A KR20197019386 A KR 20197019386A KR 102604903 B1 KR102604903 B1 KR 102604903B1
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아리 이. 프리드란드
하리하란 자야람
베레트 에탄 슈타인베르크
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에디타스 메디신, 인코포레이티드
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KR1020197019386A 2016-12-05 2017-12-05 내인성 및 공급원 DNA의 원-샷 가이드 RNA(ogRNA) 표적화를 위한 시스템 및 방법 Active KR102604903B1 (ko)

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US201662430154P 2016-12-05 2016-12-05
US62/430,154 2016-12-05
US201762503640P 2017-05-09 2017-05-09
US62/503,640 2017-05-09
PCT/US2017/064720 WO2018106693A1 (en) 2016-12-05 2017-12-05 SYSTEMS AND METHODS FOR ONE-SHOT GUIDE RNA (ogRNA) TARGETING OF ENDOGENOUS AND SOURCE DNA

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KR20190088555A KR20190088555A (ko) 2019-07-26
KR102604903B1 true KR102604903B1 (ko) 2023-11-21

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Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
CA3001711A1 (en) * 2015-10-30 2017-05-04 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US20190153440A1 (en) 2017-11-21 2019-05-23 Casebia Therapeutics Llp Materials and methods for treatment of autosomal dominant retinitis pigmentosa
WO2019118875A1 (en) * 2017-12-15 2019-06-20 Ou Li Crispr-mediated genome editing with vectors
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
WO2020055748A1 (en) * 2018-09-10 2020-03-19 The Regents Of The University Of California Modulators of cas9 polypeptides and methods of use thereof
WO2020176552A1 (en) 2019-02-25 2020-09-03 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
WO2020186059A2 (en) * 2019-03-12 2020-09-17 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
WO2020264254A1 (en) * 2019-06-28 2020-12-30 Crispr Therapeutics Ag Materials and methods for controlling gene editing
WO2021053582A1 (en) * 2019-09-18 2021-03-25 Crispr Therapeutics Ag All-in-one self inactivating crispr vectors
WO2021062201A1 (en) * 2019-09-25 2021-04-01 Spotlight Therapeutics Compositions and methods for nucleoprotein targeting and expression
US20230203220A1 (en) * 2020-05-29 2023-06-29 Regents Of The University Of Minnesota Copolymers for intracellular therapeutic nucleic acid payload delivery
CA3200815A1 (en) * 2020-12-03 2022-06-09 Benjamin OAKES Compositions and methods for the targeting of bcl11a
WO2022221741A1 (en) 2021-04-16 2022-10-20 Editas Medicine, Inc. Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)
WO2025179057A1 (en) * 2024-02-21 2025-08-28 The Regents Of The University Of California In vivo modification of cell genomes

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015070083A1 (en) 2013-11-07 2015-05-14 Editas Medicine,Inc. CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10201804973TA (en) * 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
KR102598856B1 (ko) * 2015-03-03 2023-11-07 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
WO2016176404A1 (en) * 2015-04-30 2016-11-03 The Brigham And Women's Hospital, Inc. Methods and kits for cloning-free genome editing
US20180291372A1 (en) * 2015-05-14 2018-10-11 Massachusetts Institute Of Technology Self-targeting genome editing system
CN107849547B (zh) 2015-05-16 2022-04-19 建新公司 深内含子突变的基因编辑
US11530421B2 (en) 2016-02-01 2022-12-20 The Regents Of The University Of California Self-inactivating endonuclease-encoding nucleic acids and methods of using the same
WO2017173004A1 (en) * 2016-03-30 2017-10-05 Mikuni Takayasu A method for in vivo precise genome editing

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015070083A1 (en) 2013-11-07 2015-05-14 Editas Medicine,Inc. CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
bioRxiv preprint, (201610.01. 온라인 개재) 1부.*
Nucleic Acids Research, 2014, Vol.43, No.2, pp.1297-1303 1부.*

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US20180201956A1 (en) 2018-07-19
CA3046076A1 (en) 2018-06-14
US12264331B2 (en) 2025-04-01
US9963719B1 (en) 2018-05-08
US20220017927A1 (en) 2022-01-20
US10006054B1 (en) 2018-06-26
US11028411B2 (en) 2021-06-08
US10494649B2 (en) 2019-12-03
US20200056208A1 (en) 2020-02-20
KR20190088555A (ko) 2019-07-26
JP7272952B2 (ja) 2023-05-12
US20180251792A1 (en) 2018-09-06
JP2023052089A (ja) 2023-04-11
MX2024002327A (es) 2024-03-07
US11692205B2 (en) 2023-07-04
EP3548614A1 (en) 2019-10-09
MX2019006475A (es) 2019-09-26
CN110168084A (zh) 2019-08-23
JP2019536462A (ja) 2019-12-19
WO2018106693A1 (en) 2018-06-14
AU2017373797A1 (en) 2019-06-20
US20230416787A1 (en) 2023-12-28

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