JP2017505117A5 - - Google Patents

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Publication number
JP2017505117A5
JP2017505117A5 JP2016542968A JP2016542968A JP2017505117A5 JP 2017505117 A5 JP2017505117 A5 JP 2017505117A5 JP 2016542968 A JP2016542968 A JP 2016542968A JP 2016542968 A JP2016542968 A JP 2016542968A JP 2017505117 A5 JP2017505117 A5 JP 2017505117A5
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JP
Japan
Prior art keywords
cleavage
guide rna
csy4
seq
sequence
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JP2016542968A
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English (en)
Japanese (ja)
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JP6721508B2 (ja
JP2017505117A (ja
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Priority claimed from PCT/US2014/029304 external-priority patent/WO2014144761A2/en
Priority claimed from US14/211,117 external-priority patent/US10760064B2/en
Priority claimed from PCT/US2014/035162 external-priority patent/WO2014204578A1/en
Application filed filed Critical
Priority claimed from PCT/US2014/056416 external-priority patent/WO2015099850A1/en
Publication of JP2017505117A publication Critical patent/JP2017505117A/ja
Publication of JP2017505117A5 publication Critical patent/JP2017505117A5/ja
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Publication of JP6721508B2 publication Critical patent/JP6721508B2/ja
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JP2016542968A 2013-12-26 2014-09-18 多重ガイドrna Active JP6721508B2 (ja)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201361921007P 2013-12-26 2013-12-26
US61/921,007 2013-12-26
US201461930782P 2014-01-23 2014-01-23
US61/930,782 2014-01-23
US14/211,117 2014-03-14
USPCT/US2014/028630 2014-03-14
USPCT/US2014/029068 2014-03-14
PCT/US2014/029304 WO2014144761A2 (en) 2013-03-15 2014-03-14 Increasing specificity for rna-guided genome editing
PCT/US2014/029068 WO2014144592A2 (en) 2013-03-15 2014-03-14 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
US14/211,117 US10760064B2 (en) 2013-03-15 2014-03-14 RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
PCT/US2014/028630 WO2014144288A1 (en) 2013-03-15 2014-03-14 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
USPCT/US2014/029304 2014-03-14
PCT/US2014/035162 WO2014204578A1 (en) 2013-06-21 2014-04-23 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
USPCT/US2014/035162 2014-04-23
PCT/US2014/056416 WO2015099850A1 (en) 2013-12-26 2014-09-18 Multiplex guide rnas

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019218086A Division JP7005580B2 (ja) 2013-12-26 2019-12-02 多重ガイドrna

Publications (3)

Publication Number Publication Date
JP2017505117A JP2017505117A (ja) 2017-02-16
JP2017505117A5 true JP2017505117A5 (OSRAM) 2017-10-26
JP6721508B2 JP6721508B2 (ja) 2020-07-15

Family

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JP2016542968A Active JP6721508B2 (ja) 2013-12-26 2014-09-18 多重ガイドrna
JP2019218086A Active JP7005580B2 (ja) 2013-12-26 2019-12-02 多重ガイドrna

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JP2019218086A Active JP7005580B2 (ja) 2013-12-26 2019-12-02 多重ガイドrna

Country Status (8)

Country Link
US (1) US20200165587A1 (OSRAM)
EP (2) EP3090044B1 (OSRAM)
JP (2) JP6721508B2 (OSRAM)
KR (1) KR20160102056A (OSRAM)
CN (2) CN113684205B (OSRAM)
AU (3) AU2014370416B2 (OSRAM)
CA (1) CA2935032C (OSRAM)
WO (1) WO2015099850A1 (OSRAM)

Families Citing this family (81)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9388430B2 (en) * 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
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)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
WO2015153940A1 (en) * 2014-04-03 2015-10-08 Massachusetts Institute Of Technology Methods and compositions for the production of guide rna
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP6529110B2 (ja) * 2014-12-01 2019-06-12 国立大学法人 東京大学 複数のユニットが多重に連結したdnaカセットおよび該カセットを含むベクターの製造方法
JP2018522249A (ja) 2015-04-24 2018-08-09 エディタス・メディシン、インコーポレイテッド Cas9分子/ガイドrna分子複合体の評価
EP3322804B1 (en) 2015-07-15 2021-09-01 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
CN105039309A (zh) * 2015-07-16 2015-11-11 深圳市盛捷生物技术有限公司 5’端不受碱基限制的gRNA的制备方法
AU2016316845B2 (en) 2015-08-28 2022-03-10 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
US20180327781A1 (en) * 2015-11-09 2018-11-15 Seattle Children's Hospital (dba Seattle Children's Research Institute) Novel rna-based vector system for transient and stable gene expression
EP3374494A4 (en) * 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE FOR GENE THERAPY
FI3390632T3 (fi) * 2015-12-18 2025-11-25 Danisco Us Inc Menetelmät ja koostumukset polymeraasi ii (pol-ii) -pohjaisen opas-rna:n ilmentämiseen
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
WO2018026976A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
CN106520824A (zh) * 2016-09-30 2017-03-22 北京大北农科技集团股份有限公司 多靶点编辑系统及其用途
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
KR20240064734A (ko) 2016-10-14 2024-05-13 더 제너럴 하스피탈 코포레이션 후성적으로 조절되는 부위-특이적 뉴클레아제
US12331086B2 (en) 2016-11-14 2025-06-17 Toolgen Incorporated Artificially engineered SC function control system
KR102551664B1 (ko) * 2016-12-22 2023-07-05 인텔리아 테라퓨틱스, 인크. 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
JP7219972B2 (ja) 2017-01-05 2023-02-09 ラトガース,ザ ステート ユニバーシティ オブ ニュー ジャージー Dna二本鎖切断に非依存的な標的化遺伝子編集プラットフォームおよびその用途
EP3568476A1 (en) * 2017-01-11 2019-11-20 Oxford University Innovation Limited Crispr rna
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
MX2019011272A (es) * 2017-03-22 2019-10-24 Novartis Ag Composiciones y metodos para inmunooncologia.
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
EP3612551B1 (en) 2017-04-21 2024-09-04 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2018218188A2 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Base editors with improved precision and specificity
CN108977442B (zh) * 2017-06-05 2023-01-06 广州市锐博生物科技有限公司 用于dna编辑的系统及其应用
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
WO2019161783A1 (en) * 2018-02-23 2019-08-29 Shanghaitech University Fusion proteins for base editing
CN111788307A (zh) * 2018-03-12 2020-10-16 南京北恒生物科技有限公司 工程化的嵌合向导rna及其用途
SG11202008956XA (en) 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
CN108384784A (zh) * 2018-03-23 2018-08-10 广西医科大学 一种利用CRISPR/Cas9技术敲除Endoglin基因的方法
WO2019213910A1 (en) * 2018-05-10 2019-11-14 Syngenta Participations Ag Methods and compositions for targeted editing of polynucleotides
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
EP3891281A1 (en) * 2018-12-05 2021-10-13 DSM IP Assets B.V. Crispr guide-rna expression strategies for multiplex genome engineering
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2021026318A2 (en) * 2019-08-06 2021-02-11 The Penn State Research Foundation Engineered crispr/cas9 systems for simultaneous long-term regulation of multiple targets
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP3889259A1 (en) 2020-03-30 2021-10-06 IMBA-Institut für Molekulare Biotechnologie GmbH Internal standard for crispr guide rna
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
US11643672B2 (en) 2021-10-01 2023-05-09 The Florida International University Board Of Trusters Inducible CRISPR system expression and applications thereof
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
CN114574467B (zh) * 2022-01-21 2023-05-23 华南农业大学 一种基因表达调控系统及其应用

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030198627A1 (en) * 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
EA024121B9 (ru) * 2010-05-10 2017-01-30 Дзе Реджентс Ов Дзе Юниверсити Ов Калифорния Композиции эндорибонуклеаз и способы их использования
FI3597749T3 (fi) * 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US10760064B2 (en) * 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
WO2015153940A1 (en) * 2014-04-03 2015-10-08 Massachusetts Institute Of Technology Methods and compositions for the production of guide rna

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