JP7272952B2 - 内因性および起源ＤＮＡのワンショットガイドＲＮＡ（ｏｇＲＮＡ）標的化のためのシステムおよび方法 - Google Patents

内因性および起源ＤＮＡのワンショットガイドＲＮＡ（ｏｇＲＮＡ）標的化のためのシステムおよび方法 Download PDF

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JP7272952B2
JP7272952B2 JP2019530105A JP2019530105A JP7272952B2 JP 7272952 B2 JP7272952 B2 JP 7272952B2 JP 2019530105 A JP2019530105 A JP 2019530105A JP 2019530105 A JP2019530105 A JP 2019530105A JP 7272952 B2 JP7272952 B2 JP 7272952B2
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アリ イー． フリートラント，
ハリハラン ジャヤラム，
ベレット イーサン スタインバーグ，
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エディタス・メディシン、インコーポレイテッド
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JP2019530105A 2016-12-05 2017-12-05 内因性および起源ＤＮＡのワンショットガイドＲＮＡ（ｏｇＲＮＡ）標的化のためのシステムおよび方法 Active JP7272952B2 (ja)

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JP2022208855A JP2023052089A (ja) 2016-12-05 2022-12-26 内因性および起源ＤＮＡのワンショットガイドＲＮＡ（ｏｇＲＮＡ）標的化のためのシステムおよび方法

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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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JP2022208855A Withdrawn JP2023052089A (ja) 2016-12-05 2022-12-26 内因性および起源ＤＮＡのワンショットガイドＲＮＡ（ｏｇＲＮＡ）標的化のためのシステムおよび方法

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KR (1) KR102604903B1 (https=)
CN (1) CN110168084A (https=)
AU (1) AU2017373797B2 (https=)
CA (1) CA3046076A1 (https=)
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Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
WO2017075475A1 (en) * 2015-10-30 2017-05-04 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus
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
MA50877A (fr) 2017-11-21 2020-09-30 Bayer Healthcare Llc Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante
EP3723813A4 (en) * 2017-12-15 2021-10-06 Regents of the University of Minnesota GENOMIC CRISPR MEDIATION EDITING WITH VECTORS
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED 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
EP3990636A1 (en) 2019-06-28 2022-05-04 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
EP4323522A1 (en) 2021-04-16 2024-02-21 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
MX2016007325A (es) 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
CA2963820A1 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
EP3265559B1 (en) * 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
US20190002920A1 (en) 2015-04-30 2019-01-03 The Brigham And Women's Hospital, Inc. Methods and kits for cloning-free genome editing
WO2016183438A1 (en) 2015-05-14 2016-11-17 Massachusetts Institute Of Technology Self-targeting genome editing system
WO2016186772A2 (en) 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
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
LIU, K.I., et al.，"A chemical-inducible CRISPR-Cas9 system for rapid control of genome editing."，NATURE CHEMICAL BIOLOGY，2016年09月12日，Vol.12, No.11，pp.980-987+Online Methods+Supplementary，DOI: 10.1038/nchembio.2179，[Published online]
MOORE, R., et al.，"CRISPR-based self-cleaving mechanism for cotrollable gene delivery in human cells."，NUCLEIC ACIDS RESEARCH，2014年12月18日，Vol.43, No.2，pp.1297-1303，DOI: 10.1093/nar/gku1326，[Published online]

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KR20190088555A (ko) 2019-07-26
US20180251792A1 (en) 2018-09-06
US20200056208A1 (en) 2020-02-20
US10494649B2 (en) 2019-12-03
AU2017373797B2 (en) 2024-08-29
US11028411B2 (en) 2021-06-08
CN110168084A (zh) 2019-08-23
MX2019006475A (es) 2019-09-26
WO2018106693A1 (en) 2018-06-14
AU2017373797A1 (en) 2019-06-20
JP2023052089A (ja) 2023-04-11
KR102604903B1 (ko) 2023-11-21
US20220017927A1 (en) 2022-01-20
US11692205B2 (en) 2023-07-04
MX2024002327A (es) 2024-03-07
US12264331B2 (en) 2025-04-01
JP2019536462A (ja) 2019-12-19
EP3548614A1 (en) 2019-10-09
US10006054B1 (en) 2018-06-26
US20180201956A1 (en) 2018-07-19
US9963719B1 (en) 2018-05-08
CA3046076A1 (en) 2018-06-14
US20230416787A1 (en) 2023-12-28

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