JP2019524149A - 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 - Google Patents

一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 Download PDF

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JP2019524149A
JP2019524149A JP2019510339A JP2019510339A JP2019524149A JP 2019524149 A JP2019524149 A JP 2019524149A JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019524149 A JP2019524149 A JP 2019524149A
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sequence
seq
snp
mutation
pam
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JP2019524149A5 (cg-RX-API-DMAC7.html
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タラ ムーア
タラ ムーア
アンドリュー ネスビット
アンドリュー ネスビット
デイビッド コートニー
デイビッド コートニー
ケイティー クリスティー
ケイティー クリスティー
ジーン リー
ジーン リー
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Avellino Lab USA Inc
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Avellino Lab USA Inc
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Publication of JP2019524149A publication Critical patent/JP2019524149A/ja
Publication of JP2019524149A5 publication Critical patent/JP2019524149A5/ja
Priority to JP2021214009A priority Critical patent/JP2022046694A/ja
Priority to JP2024002942A priority patent/JP2024041905A/ja
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    • C12N15/09—Recombinant DNA-technology
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
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    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
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    • C12N9/14—Hydrolases (3)
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    • A—HUMAN NECESSITIES
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JP2019510339A 2016-08-20 2017-08-21 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 Pending JP2019524149A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021214009A JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2024002942A JP2024041905A (ja) 2016-08-20 2024-01-12 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662377586P 2016-08-20 2016-08-20
US62/377,586 2016-08-20
US201762462808P 2017-02-23 2017-02-23
US62/462,808 2017-02-23
US201762501750P 2017-05-05 2017-05-05
US62/501,750 2017-05-05
PCT/US2017/047861 WO2018039145A1 (en) 2016-08-20 2017-08-21 Single guide rna, crispr/cas9 systems, and methods of use thereof

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JP2021214009A Division JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

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JP2019524149A true JP2019524149A (ja) 2019-09-05
JP2019524149A5 JP2019524149A5 (cg-RX-API-DMAC7.html) 2020-10-01

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JP2019510339A Pending JP2019524149A (ja) 2016-08-20 2017-08-21 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2021214009A Pending JP2022046694A (ja) 2016-08-20 2021-12-28 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JP2024002942A Pending JP2024041905A (ja) 2016-08-20 2024-01-12 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

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JP2024002942A Pending JP2024041905A (ja) 2016-08-20 2024-01-12 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法

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US (1) US20190185850A1 (cg-RX-API-DMAC7.html)
EP (1) EP3500677A4 (cg-RX-API-DMAC7.html)
JP (3) JP2019524149A (cg-RX-API-DMAC7.html)
KR (2) KR20230155013A (cg-RX-API-DMAC7.html)
CN (1) CN109963945A (cg-RX-API-DMAC7.html)
WO (1) WO2018039145A1 (cg-RX-API-DMAC7.html)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPWO2018225807A1 (ja) * 2017-06-07 2020-04-09 国立大学法人 東京大学 顆粒状角膜変性症に対する遺伝子治療薬
JP2022046694A (ja) * 2016-08-20 2022-03-23 アベリノ ラボ ユーエスエー インコーポレイテッド 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
US11987809B2 (en) 2015-11-13 2024-05-21 Avellino Lab Usa, Inc. Methods for the treatment of corneal dystrophies

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
SG11201803173VA (en) 2015-10-23 2018-05-30 Harvard College Nucleobase editors and uses thereof
EP4644567A2 (en) 2015-11-30 2025-11-05 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2020225754A1 (en) * 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
WO2019165322A1 (en) * 2018-02-22 2019-08-29 Moore Tara Crispr/cas9 systems, and methods of use thereof
WO2020046861A1 (en) * 2018-08-27 2020-03-05 Avellino Lab Usa, Inc. Crispr/cas9 systems, and methods of use thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, 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
MA52074A (fr) * 2018-03-19 2021-01-27 Bayer Healthcare Llc Nouveaux systèmes d'endonucléase à arn programmable et leurs utilisations
CN112567030A (zh) * 2018-05-08 2021-03-26 勒芬天主教大学 生物传感器
US12157760B2 (en) 2018-05-23 2024-12-03 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
JP7564102B2 (ja) 2018-11-28 2024-10-08 クリスパー セラピューティクス アクチェンゲゼルシャフト ＬＮＰでの使用に最適化された、ＣＡＳ９をコードするｍＲＮＡ
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst Inc Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110499335B (zh) * 2019-08-08 2023-03-28 复旦大学 CRISPR/SauriCas9基因编辑系统及其应用
CN110551763B (zh) * 2019-08-08 2023-03-10 复旦大学 CRISPR/SlutCas9基因编辑系统及其应用
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
JP2023505737A (ja) * 2019-12-11 2023-02-10 アベリノ ラボ ユーエスエー インコーポレイテッド Ｒ１２４ｈ突然変異を備えたトランスフォーミング増殖因子ベータ誘導遺伝子の短鎖干渉ｒｎａを使用したアレル特異的サイレンシング
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
CN111926015B (zh) * 2020-08-24 2022-07-15 武汉纽福斯生物科技有限公司 寡核苷酸、病毒载体及其应用和RNAi药物制剂
EP4288541A1 (en) * 2021-02-05 2023-12-13 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2023034704A1 (en) * 2021-08-31 2023-03-09 The Regents Of The University Of California Products and methods for annotating gene function using locally haploid, human non-cancer cells
EP4469577A2 (en) * 2022-01-26 2024-12-04 Orthobio Therapeutics, Inc. Gene editing to improve joint function
US20250235559A1 (en) * 2022-02-25 2025-07-24 Incisive Genetics Inc. Gene editing reporter system and guide rna and composition related thereto; composition and method for knocking out dna with more than two grnas; gene editing in the eye; and gene editing using base editors
CN115948537B (zh) * 2022-12-19 2024-04-09 湖南家辉生物技术有限公司 一种基因chst3复合杂合突变的应用及检测试剂和应用
CN116286823A (zh) * 2023-04-04 2023-06-23 尧唐(上海)生物科技有限公司 靶向TGFBI基因的sgRNA、载体及其应用
WO2025043140A1 (en) * 2023-08-23 2025-02-27 Emendobio Inc. Methods and compositions for modifying expression of a mutant transforming growth factor beta induced (tgfbi) allele
CN119464347B (zh) * 2024-11-26 2025-10-03 扬州大学 基于CRISPR-Cas9系统的噬菌体基因组编辑载体及其编辑方法和应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015523856A (ja) * 2012-05-25 2015-08-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフ Ｒｎａ依存性標的ｄｎａ修飾およびｒｎａ依存性転写調節のための方法および組成物
JP2016502840A (ja) * 2012-12-06 2016-02-01 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ．， ＬＬＣ Ｃｒｉｓｐｒに基づくゲノム修飾および制御

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US9895451B2 (en) 2011-12-02 2018-02-20 Yale University Formulations for targeted release of agents to low pH tissue environments or cellular compartments and methods of use thereof
SG11201500852WA (en) 2012-08-29 2015-04-29 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
KR20150105635A (ko) 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP2896697B1 (en) 2012-12-12 2015-09-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
PL2771468T3 (pl) 2012-12-12 2015-07-31 Broad Inst Inc Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami
US20140186919A1 (en) 2012-12-12 2014-07-03 Feng Zhang Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
JP6414945B2 (ja) * 2013-03-11 2018-10-31 Ｊｃｒファーマ株式会社 ヒト角膜上皮シートの製造法
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
CN106062197A (zh) * 2013-06-17 2016-10-26 布罗德研究所有限公司 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化
SG11201600059QA (en) * 2013-07-09 2016-02-26 Harvard College Multiplex rna-guided genome engineering
WO2015021426A1 (en) * 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
US10787684B2 (en) * 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
US11473084B2 (en) * 2015-10-09 2022-10-18 The Children's Hospital Of Philadelphia Compositions and methods for treating Huntington's disease and related disorders
WO2017083852A1 (en) * 2015-11-13 2017-05-18 MOORE, Tara Methods for the treatment of corneal dystrophies
KR20230155013A (ko) * 2016-08-20 2023-11-09 아벨리노 랩 유에스에이, 인크. 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015523856A (ja) * 2012-05-25 2015-08-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフ Ｒｎａ依存性標的ｄｎａ修飾およびｒｎａ依存性転写調節のための方法および組成物
JP2016502840A (ja) * 2012-12-06 2016-02-01 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ．， ＬＬＣ Ｃｒｉｓｐｒに基づくゲノム修飾および制御

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GENE THERAPY, vol. 23, JPN6021029995, August 2015 (2015-08-01), pages 108 - 112, ISSN: 0004958737 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11987809B2 (en) 2015-11-13 2024-05-21 Avellino Lab Usa, Inc. Methods for the treatment of corneal dystrophies
JP2022046694A (ja) * 2016-08-20 2022-03-23 アベリノ ラボ ユーエスエー インコーポレイテッド 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法
JPWO2018225807A1 (ja) * 2017-06-07 2020-04-09 国立大学法人 東京大学 顆粒状角膜変性症に対する遺伝子治療薬
JP7161730B2 (ja) 2017-06-07 2022-10-27 国立大学法人 東京大学 顆粒状角膜変性症に対する遺伝子治療薬

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