JP2023088984A5 - - Google Patents

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Publication number
JP2023088984A5
JP2023088984A5 JP2023045024A JP2023045024A JP2023088984A5 JP 2023088984 A5 JP2023088984 A5 JP 2023088984A5 JP 2023045024 A JP2023045024 A JP 2023045024A JP 2023045024 A JP2023045024 A JP 2023045024A JP 2023088984 A5 JP2023088984 A5 JP 2023088984A5
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JP
Japan
Prior art keywords
effector protein
sequence
cas13b effector
cas13b
target rna
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Pending
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JP2023045024A
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English (en)
Japanese (ja)
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JP2023088984A (ja
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Priority claimed from PCT/US2018/022751 external-priority patent/WO2018170333A1/en
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Publication of JP2023088984A publication Critical patent/JP2023088984A/ja
Publication of JP2023088984A5 publication Critical patent/JP2023088984A5/ja
Pending legal-status Critical Current

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JP2023045024A 2017-03-15 2023-03-22 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系 Pending JP2023088984A (ja)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201762471710P 2017-03-15 2017-03-15
US62/471,710 2017-03-15
US201762566829P 2017-10-02 2017-10-02
US62/566,829 2017-10-02
PCT/US2018/022751 WO2018170333A1 (en) 2017-03-15 2018-03-15 Novel cas13b orthologues crispr enzymes and systems
JP2019551295A JP2020511141A (ja) 2017-03-15 2018-03-15 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系

Related Parent Applications (1)

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JP2019551295A Division JP2020511141A (ja) 2017-03-15 2018-03-15 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系

Publications (2)

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JP2023088984A JP2023088984A (ja) 2023-06-27
JP2023088984A5 true JP2023088984A5 (https=) 2025-07-25

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ID=61874014

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JP2019551295A Pending JP2020511141A (ja) 2017-03-15 2018-03-15 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系
JP2023045024A Pending JP2023088984A (ja) 2017-03-15 2023-03-22 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系

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JP2019551295A Pending JP2020511141A (ja) 2017-03-15 2018-03-15 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系

Country Status (7)

Country Link
US (2) US11739308B2 (https=)
EP (2) EP3596207B1 (https=)
JP (2) JP2020511141A (https=)
KR (2) KR102454284B1 (https=)
CN (2) CN110959039B (https=)
AU (3) AU2018234825B2 (https=)
WO (1) WO2018170333A1 (https=)

Families Citing this family (66)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116814590A (zh) * 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
US11104937B2 (en) 2017-03-15 2021-08-31 The Broad Institute, Inc. CRISPR effector system based diagnostics
JP2020511141A (ja) 2017-03-15 2020-04-16 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系
US11174515B2 (en) 2017-03-15 2021-11-16 The Broad Institute, Inc. CRISPR effector system based diagnostics
US11963966B2 (en) 2017-03-31 2024-04-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
US12590288B2 (en) 2017-04-12 2026-03-31 The Broad Institute, Inc. Method of treating an inflammatory disease by administering an agent which binds a surface receptor on a tuft cell that induces an ILC class 2 inflammatory response
AU2018251801B2 (en) * 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
EP3622092A4 (en) 2017-05-11 2021-06-23 The Broad Institute, Inc. METHODS AND COMPOSITIONS OF USE OF CD8 + TUMOR-INFILTRATING LYMPHOCYTE SUBTYPES AND GENE SIGNATURES THEREOF
WO2018232195A1 (en) 2017-06-14 2018-12-20 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
KR102761791B1 (ko) * 2017-06-26 2025-02-05 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법
US12105089B2 (en) 2017-07-17 2024-10-01 The Broad Institute, Inc. Cell atlas of the healthy and ulcerative colitis human colon
AU2018338318B2 (en) * 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
WO2019071048A1 (en) * 2017-10-04 2019-04-11 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION
US20200332272A1 (en) * 2017-10-23 2020-10-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2019084062A1 (en) * 2017-10-23 2019-05-02 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION
WO2019094955A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas
WO2019113506A1 (en) 2017-12-07 2019-06-13 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
AU2019213047B2 (en) * 2018-01-29 2025-07-24 Massachusetts Institute Of Technology CRISPR effector system based diagnostics
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
SG11202012015YA (en) * 2018-06-08 2021-01-28 Locanabio Inc Compositions and methods for the modulation of adaptive immunity
KR20210058806A (ko) 2018-06-08 2021-05-24 로카나바이오 인크. Rna 표적화 융합 단백질 조성물 및 사용 방법
US12036240B2 (en) 2018-06-14 2024-07-16 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
WO2020006036A1 (en) 2018-06-26 2020-01-02 Massachusetts Institute Of Technology Crispr effector system based amplification methods, systems, and diagnostics
AU2019291827A1 (en) 2018-06-26 2020-12-24 Massachusetts Institute Of Technology Crispr double nickase based amplification compositions, systems, and methods
EP3830301B1 (en) 2018-08-01 2024-05-22 Mammoth Biosciences, Inc. Programmable nuclease compositions and methods of use thereof
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
EP3864152B1 (en) * 2018-10-12 2023-08-16 Peking University Methods and compositions for editing rnas
WO2020097445A1 (en) 2018-11-09 2020-05-14 Inari Agriculture, Inc. Rna-guided nucleases and dna binding proteins
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
US20250043366A1 (en) 2018-12-13 2025-02-06 The Broad Institute, Inc. Tiled assays using crispr-cas based detection
EP3906308A1 (en) * 2018-12-31 2021-11-10 Research Institute at Nationwide Children's Hospital Dux4 rna silencing using rna targeting crispr-cas13b
EP3931313A2 (en) 2019-01-04 2022-01-05 Mammoth Biosciences, Inc. Programmable nuclease improvements and compositions and methods for nucleic acid amplification and detection
US20200385713A1 (en) 2019-01-22 2020-12-10 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
EP3914260A4 (en) 2019-01-22 2023-05-17 Korro Bio, Inc. RNA-EDITING OLIGONUCLEOTIDES AND THEIR USE
CA3127243A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
US20220154258A1 (en) 2019-03-14 2022-05-19 The Broad Institute, Inc. Crispr effector system based multiplex diagnostics
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
WO2021011493A1 (en) * 2019-07-12 2021-01-21 Duke University 3' utr crispr-dcas 13 engineering system and methods of using same
US20220333208A1 (en) 2019-09-03 2022-10-20 The Broad Institute, Inc. Crispr effector system based multiplex cancer diagnostics
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
CN115175996A (zh) * 2019-09-20 2022-10-11 博德研究所 新颖vi型crispr酶和系统
US11844800B2 (en) 2019-10-30 2023-12-19 Massachusetts Institute Of Technology Methods and compositions for predicting and preventing relapse of acute lymphoblastic leukemia
WO2021146641A1 (en) 2020-01-17 2021-07-22 The Broad Institute, Inc. Small type ii-d cas proteins and methods of use thereof
CN115315519A (zh) * 2020-02-28 2022-11-08 辉大(上海)生物科技有限公司 VI-E型和VI-F型CRISPR-Cas系统及其用途
WO2021188734A1 (en) 2020-03-17 2021-09-23 The Broad Institute, Inc. Crispr system high throughput diagnostic systems and methods
WO2021195519A1 (en) * 2020-03-27 2021-09-30 University Of Rochester Targeted destruction of viral rna by crispr-cas13
HRP20240099T8 (hr) * 2020-04-08 2024-06-21 Elisabeth ZEISBERG Sve-u-jedan aav vektori za liječenje koronavirus-induciranih bolesti
CN112430586B (zh) * 2020-11-16 2021-09-07 珠海舒桐医疗科技有限公司 一种VI-B型CRISPR/Cas13基因编辑系统及其应用
WO2022136370A1 (en) * 2020-12-22 2022-06-30 Helmholtz Zentrum Muenchen - Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Application of crispr/cas13 for therapy of rna virus and/or bacterium induced diseases
WO2022182863A1 (en) * 2021-02-24 2022-09-01 University Of Massachusetts Inducible single aav system and uses thereof
EP4347818A2 (en) 2021-06-01 2024-04-10 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof
CN115678872A (zh) * 2021-06-04 2023-02-03 中国科学院脑科学与智能技术卓越创新中心 新型Cas13蛋白及其筛选方法和应用
CN113832180B (zh) * 2021-08-03 2024-06-28 华中农业大学 CRISPR/Cas13b介导的棉花RNA转录调控方法
EP4419686A4 (en) * 2021-10-21 2025-10-15 Nanothera Biosciences Inc COMPOSITIONS AND METHODS OF EXTINGUISHING KRAS
WO2023081902A1 (en) * 2021-11-05 2023-05-11 University Of Florida Research Foundation, Inc. Systems and methods for target polynucleotide detection with crispr/cas12a using activators
WO2023220566A1 (en) * 2022-05-09 2023-11-16 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
CN116254297A (zh) * 2022-07-11 2023-06-13 华中科技大学 一种基于Cas13的可诱导基因敲低的单质粒载体及应用
GB202214015D0 (en) * 2022-09-26 2022-11-09 Univ Oxford Innovation Ltd RNA editing vector
WO2024112957A1 (en) 2022-11-23 2024-05-30 Amber Bio Inc. Gene-modifying endonucleases
CN120548359A (zh) * 2023-01-18 2025-08-26 国家科学技术发展局 具有提高的效率的突变的Cas13b
KR20250151430A (ko) 2023-02-15 2025-10-21 아버 바이오테크놀로지스, 인크. 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법
US20250163395A1 (en) * 2023-11-21 2025-05-22 Roche Molecular Systems, Inc. Systems and methods of novel crispr-cas proteins for in-vitro applications
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining
WO2026015829A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Small reverse transcriptases and gene editing systems comprising such
WO2026015832A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Reverse transcriptases and gene editing systems comprising such

Family Cites Families (107)

* 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
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
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
DE122007000007I2 (de) 1986-04-09 2010-12-30 Genzyme Corp Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
GB9114259D0 (en) 1991-07-02 1991-08-21 Ici Plc Plant derived enzyme and dna sequences
JP2952041B2 (ja) 1992-07-27 1999-09-20 パイオニア ハイ−ブレッド インターナショナル，インコーポレイテッド 培養ダイズ細胞のａｇｒｏｂａｃｔｅｒｉｕｍ媒介形質転換の改良法
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US5789156A (en) 1993-06-14 1998-08-04 Basf Ag Tetracycline-regulated transcriptional inhibitors
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US6007845A (en) 1994-07-22 1999-12-28 Massachusetts Institute Of Technology Nanoparticles and microparticles of non-linear hydrophilic-hydrophobic multiblock copolymers
AU698739B2 (en) 1995-06-06 1998-11-05 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5985662A (en) 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
US5985309A (en) 1996-05-24 1999-11-16 Massachusetts Institute Of Technology Preparation of particles for inhalation
US5855913A (en) 1997-01-16 1999-01-05 Massachusetts Instite Of Technology Particles incorporating surfactants for pulmonary drug delivery
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
GB9907461D0 (en) 1999-03-31 1999-05-26 King S College London Neurite regeneration
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
EP1025217B1 (en) 1997-10-24 2006-10-04 Invitrogen Corporation Recombinational cloning using nucleic acids having recombination sites
US6750059B1 (en) 1998-07-16 2004-06-15 Whatman, Inc. Archiving of vectors
GB0024550D0 (https=) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7776321B2 (en) 2001-09-26 2010-08-17 Mayo Foundation For Medical Education And Research Mutable vaccines
GB0125216D0 (en) 2001-10-19 2001-12-12 Univ Strathclyde Dendrimers for use in targeted delivery
CN1620508A (zh) 2001-12-21 2005-05-25 牛津生物医学（英国）有限公司 转基因生物
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
WO2004015075A2 (en) 2002-08-08 2004-02-19 Dharmacon, Inc. Short interfering rnas having a hairpin structure containing a non-nucleotide loop
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
EP1558724A4 (en) 2002-11-01 2006-08-02 New England Biolabs Inc ORGANIC RNA SCREENING AND ITS USE IN INTERRUPTING GENE TRANSMISSION IN THE ENVIRONMENT
CA2532228C (en) 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
EP1664316B1 (en) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
GB0325379D0 (en) 2003-10-30 2003-12-03 Oxford Biomedica Ltd Vectors
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
AU2005274948B2 (en) 2004-07-16 2011-09-22 Genvec, Inc. Vaccines against aids comprising CMV/R-nucleic acid constructs
GB0422877D0 (en) 2004-10-14 2004-11-17 Univ Glasgow Bioactive polymers
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
US8044019B2 (en) 2005-10-28 2011-10-25 Mitsubishi Tanabe Pharma Corporation Cell penetrating peptide
CN101346393B (zh) 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
JP2008078613A (ja) 2006-08-24 2008-04-03 Rohm Co Ltd 窒化物半導体の製造方法及び窒化物半導体素子
US20100099858A1 (en) 2006-09-28 2010-04-22 Mirkin Chad A Maximizing Oligonucleotide Loading on Gold Nanoparticle
WO2008064289A2 (en) 2006-11-21 2008-05-29 The Samuel Roberts Noble Foundation, Inc. Biofuel production methods and compositions
WO2008149176A1 (en) 2007-06-06 2008-12-11 Cellectis Meganuclease variants cleaving a dna target sequence from the mouse rosa26 locus and uses thereof
US8404658B2 (en) 2007-12-31 2013-03-26 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
WO2009147368A1 (en) 2008-06-04 2009-12-10 Medical Research Council Peptides
BRPI0910198A2 (pt) 2008-06-30 2016-01-12 Silenseed Ltd composição para administração a partir de um sítio local de um agente baseado em nucleotídeos e método para tratar um sujeito sofrendo de uma doença requerendo tratamento local com um agente baseado em nucleotídeo.
US20110117189A1 (en) 2008-07-08 2011-05-19 S.I.F.I. Societa' Industria Farmaceutica Italiana S.P.A. Ophthalmic compositions for treating pathologies of the posterior segment of the eye
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
GB2465749B (en) 2008-11-25 2013-05-08 Algentech Sas Plant cell transformation method
WO2010096488A1 (en) 2009-02-17 2010-08-26 The Regents Of The University Of California Method of reducing acetylation in plants to improve biofuel production
EP2449106B1 (en) 2009-07-01 2015-04-08 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
EP2454371B1 (en) 2009-07-13 2021-01-20 Somagenics, Inc. Chemical modification of small hairpin rnas for inhibition of gene expression
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US20120003201A1 (en) 2010-04-21 2012-01-05 Nicholas Susanne B Vault agents for chronic kidney disease
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
US8372951B2 (en) 2010-05-14 2013-02-12 National Tsing Hua University Cell penetrating peptides for intracellular delivery
WO2011148194A1 (en) 2010-05-28 2011-12-01 Oxford Biomedica (Uk) Ltd Delivery of lentiviral vectors to the brain
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
EP2691443B1 (en) 2011-03-28 2021-02-17 Massachusetts Institute of Technology Conjugated lipomers and uses thereof
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US20120295960A1 (en) 2011-05-20 2012-11-22 Oxford Biomedica (Uk) Ltd. Treatment regimen for parkinson's disease
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
US20130185823A1 (en) 2012-01-16 2013-07-18 Academia Sinica Mesoporous silica nanoparticle-mediated delivery of dna into arabidopsis root
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
US8614194B1 (en) 2012-07-25 2013-12-24 Kaohsiung Medical University Anionic cell penetrating peptide and its use for intracellular delivery
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
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
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
RU2716420C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
WO2014204723A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions
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
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
MX374532B (es) 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
JP7103750B2 (ja) * 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
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
WO2015089427A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
JP6712948B2 (ja) * 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
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
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
JP2018515142A (ja) 2015-05-15 2018-06-14 ダーマコン，インコーポレイテッド． Ｃａｓ９介在遺伝子編集用の合成シングルガイドｒｎａ
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016279077A1 (en) * 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
CN109642231A (zh) * 2016-06-17 2019-04-16 博德研究所 Vi型crispr直向同源物和系统
US12404514B2 (en) 2016-12-09 2025-09-02 The Broad Institute, Inc. CRISPR-systems for modifying a trait of interest in a plant
JP2020511141A (ja) 2017-03-15 2020-04-16 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系

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