JP6799058B2 - アレル選択的な遺伝子編集およびその使用 - Google Patents

アレル選択的な遺伝子編集およびその使用 Download PDF

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JP6799058B2
JP6799058B2 JP2018515082A JP2018515082A JP6799058B2 JP 6799058 B2 JP6799058 B2 JP 6799058B2 JP 2018515082 A JP2018515082 A JP 2018515082A JP 2018515082 A JP2018515082 A JP 2018515082A JP 6799058 B2 JP6799058 B2 JP 6799058B2
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nucleotides
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monomer
gene
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JP2018527013A (ja
JP2018527013A5 (https=
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パドマナブ・チブクラ
グランサム レイチェル・ウィルキー−
グランサム レイチェル・ウィルキー−
キヨシ・タチカワ
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Arcturus Therapeutics Inc
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N2310/00—Structure or type of the nucleic acid
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JP2018515082A 2015-09-21 2016-09-21 アレル選択的な遺伝子編集およびその使用 Active JP6799058B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562221407P 2015-09-21 2015-09-21
US62/221,407 2015-09-21
US201662330827P 2016-05-02 2016-05-02
US62/330,827 2016-05-02
PCT/US2016/052889 WO2017053431A2 (en) 2015-09-21 2016-09-21 Allele selective gene editing and uses thereof

Publications (3)

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JP2018527013A JP2018527013A (ja) 2018-09-20
JP2018527013A5 JP2018527013A5 (https=) 2019-10-31
JP6799058B2 true JP6799058B2 (ja) 2020-12-09

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US (1) US10369232B2 (https=)
EP (1) EP3353298B1 (https=)
JP (1) JP6799058B2 (https=)
CN (1) CN108350454B (https=)
ES (1) ES2959815T3 (https=)
WO (1) WO2017053431A2 (https=)

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3690044B1 (en) 2014-02-11 2024-01-10 The Regents of the University of Colorado, a body corporate Crispr enabled multiplexed genome engineering
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
CA2970683A1 (en) 2014-12-18 2016-06-23 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
WO2017004261A1 (en) 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
AU2017280353B2 (en) 2016-06-24 2021-11-11 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
KR20260019012A (ko) 2016-10-07 2026-02-09 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Ｓ. 피오게네스 ｃａｓ９ 돌연변이 유전자 및 이에 의해 암호화되는 폴리펩티드
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
KR20190082318A (ko) 2016-11-22 2019-07-09 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Crispr/cpf1 시스템 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
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
US12390514B2 (en) 2017-03-09 2025-08-19 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
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
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
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
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
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
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
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
JP2020536501A (ja) * 2017-09-28 2020-12-17 ツールゲン インコーポレイテッドＴｏｏｌｇｅｎ Ｉｎｃｏｒｐｏｒａｔｅｄ 遺伝子発現調節のための人為的なゲノム操作
US20190098879A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. Non-Human Animals Comprising A Humanized TTR Locus And Methods Of Use
EP3688161A1 (en) * 2017-09-29 2020-08-05 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating attr amyloidosis
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US10227576B1 (en) 2018-06-13 2019-03-12 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
US12227776B2 (en) 2018-06-13 2025-02-18 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
EP3908257A4 (en) * 2019-01-08 2022-11-09 Asklepios Biopharmaceutical, Inc. METHODS OF TREATMENT OF CYSTIC FIBROSIS
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
GB201901873D0 (en) * 2019-02-11 2019-04-03 Proqr Therapeutics Ii Bv Antisense oligonucleotides for nucleic acid editing
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, 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
US11891618B2 (en) 2019-06-04 2024-02-06 Regeneron Pharmaceuticals, Inc. Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
WO2021050593A1 (en) 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
WO2025166325A1 (en) * 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101629017B1 (ko) * 2007-05-22 2016-06-10 아크투루스 쎄라퓨틱스, 인크. 히드록시메틸 치환된 ｒｎａ 올리고뉴클레오티드 및 ｒｎａ 복합체
AU2013293270B2 (en) * 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
IL239344B2 (en) * 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
US9957515B2 (en) * 2013-03-15 2018-05-01 Cibus Us Llc Methods and compositions for targeted gene modification
CN105683376A (zh) * 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
CN105683379A (zh) * 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
WO2014204724A1 (en) * 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
EP3470089A1 (en) * 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components

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WO2017053431A3 (en) 2017-06-01
ES2959815T3 (es) 2024-02-28
WO2017053431A2 (en) 2017-03-30
CN108350454A (zh) 2018-07-31
JP2018527013A (ja) 2018-09-20
CA3002647A1 (en) 2017-03-30
CN108350454B (zh) 2022-05-10
EP3353298A4 (en) 2019-09-25
US20170080107A1 (en) 2017-03-23
EP3353298B1 (en) 2023-09-13
EP3353298A2 (en) 2018-08-01
US10369232B2 (en) 2019-08-06

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