JP2021532815A5 - - Google Patents

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Publication number
JP2021532815A5
JP2021532815A5 JP2021506471A JP2021506471A JP2021532815A5 JP 2021532815 A5 JP2021532815 A5 JP 2021532815A5 JP 2021506471 A JP2021506471 A JP 2021506471A JP 2021506471 A JP2021506471 A JP 2021506471A JP 2021532815 A5 JP2021532815 A5 JP 2021532815A5
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JP
Japan
Prior art keywords
cas12b
cell
target
protein
oligonucleotide
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Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
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JP2021506471A
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English (en)
Japanese (ja)
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JP2021532815A (ja
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Priority claimed from PCT/US2019/045582 external-priority patent/WO2020033601A1/en
Publication of JP2021532815A publication Critical patent/JP2021532815A/ja
Publication of JP2021532815A5 publication Critical patent/JP2021532815A5/ja
Priority to JP2024095510A priority Critical patent/JP2024138266A/ja
Pending legal-status Critical Current

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JP2021506471A 2018-08-07 2019-08-07 新規Cas12b酵素およびシステム Pending JP2021532815A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024095510A JP2024138266A (ja) 2018-08-07 2024-06-13 新規Cas12b酵素およびシステム

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201862715640P 2018-08-07 2018-08-07
US62/715,640 2018-08-07
US201862744080P 2018-10-10 2018-10-10
US62/744,080 2018-10-10
US201862751196P 2018-10-26 2018-10-26
US62/751,196 2018-10-26
US201962794929P 2019-01-21 2019-01-21
US62/794,929 2019-01-21
US201962831028P 2019-04-08 2019-04-08
US62/831,028 2019-04-08
PCT/US2019/045582 WO2020033601A1 (en) 2018-08-07 2019-08-07 Novel cas12b enzymes and systems

Related Child Applications (1)

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JP2024095510A Division JP2024138266A (ja) 2018-08-07 2024-06-13 新規Cas12b酵素およびシステム

Publications (2)

Publication Number Publication Date
JP2021532815A JP2021532815A (ja) 2021-12-02
JP2021532815A5 true JP2021532815A5 (enExample) 2022-08-03

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JP2021506471A Pending JP2021532815A (ja) 2018-08-07 2019-08-07 新規Cas12b酵素およびシステム
JP2024095510A Pending JP2024138266A (ja) 2018-08-07 2024-06-13 新規Cas12b酵素およびシステム

Family Applications After (1)

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JP2024095510A Pending JP2024138266A (ja) 2018-08-07 2024-06-13 新規Cas12b酵素およびシステム

Country Status (8)

Country Link
US (1) US20210163944A1 (enExample)
EP (1) EP3833761A1 (enExample)
JP (2) JP2021532815A (enExample)
KR (1) KR20210056329A (enExample)
CN (1) CN113286884A (enExample)
AU (2) AU2019318079B2 (enExample)
CA (1) CA3106035A1 (enExample)
WO (1) WO2020033601A1 (enExample)

Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
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
WO2019126709A1 (en) * 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
SG11202102068TA (en) * 2018-07-31 2021-03-30 Broad Inst Inc Novel crispr enzymes and systems
CN119264211A (zh) 2018-08-27 2025-01-07 瑞泽恩制药公司 拉曼光谱在下游纯化中的应用
CN109337904B (zh) * 2018-11-02 2020-12-25 中国科学院动物研究所 基于C2c1核酸酶的基因组编辑系统和方法
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
US12421557B2 (en) 2019-08-16 2025-09-23 The Broad Institute, Inc. Methods for predicting outcomes and treating colorectal cancer using a cell atlas
US12264367B2 (en) 2019-09-19 2025-04-01 The Broad Institute, Inc. Methods of in vivo evaluation of gene function
US12195725B2 (en) 2019-10-03 2025-01-14 Dana-Farber Cancer Institute, Inc. Compositions and methods for modulating and detecting tissue specific TH17 cell pathogenicity
US11981922B2 (en) 2019-10-03 2024-05-14 Dana-Farber Cancer Institute, Inc. Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment
US11793787B2 (en) 2019-10-07 2023-10-24 The Broad Institute, Inc. Methods and compositions for enhancing anti-tumor immunity by targeting steroidogenesis
US11844800B2 (en) 2019-10-30 2023-12-19 Massachusetts Institute Of Technology Methods and compositions for predicting and preventing relapse of acute lymphoblastic leukemia
US12195723B2 (en) 2019-11-08 2025-01-14 The Broad Institute, Inc. Engineered antigen presenting cells and uses thereof
US12241830B2 (en) 2019-12-06 2025-03-04 Broad Institute, Inc. Living biosensors
WO2021146641A1 (en) 2020-01-17 2021-07-22 The Broad Institute, Inc. Small type ii-d cas proteins and methods of use thereof
US12165747B2 (en) 2020-01-23 2024-12-10 The Broad Institute, Inc. Molecular spatial mapping of metastatic tumor microenvironment
US20230071360A1 (en) * 2020-02-24 2023-03-09 Chan Zuckerberg Biohub, Inc. Nucleic acid sequence detection by measuring free monoribonucleotides generated by endonuclease collateral cleavage activity
CN111349649B (zh) * 2020-03-16 2020-11-17 三峡大学 一种用于双孢蘑菇的基因编辑的方法及应用
EP4132951A4 (en) * 2020-04-09 2025-06-11 Verve Therapeutics, Inc. CHEMICALLY MODIFIED GENE EDITING GUIDE RNAS WITH CAS12B
WO2022046667A1 (en) * 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cells and non-human animals engineered to express adar1 and uses thereof
WO2022040909A1 (en) * 2020-08-25 2022-03-03 Institute Of Zoology, Chinese Academy Of Sciences Split cas12 systems and methods of use thereof
JP2023543360A (ja) * 2020-09-29 2023-10-13 ニューエクセル・セラピューティクス・インコーポレイテッド Dlx2ベクター
AU2021391783A1 (en) * 2020-12-03 2023-06-22 Scribe Therapeutics Inc. Compositions and methods for the targeting of bcl11a
CN113308451B (zh) * 2020-12-07 2023-07-25 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法
US20240327933A1 (en) * 2020-12-16 2024-10-03 The Broad Institute, Inc. Coronavirus rapid diagnostics
CN112538500A (zh) * 2020-12-25 2021-03-23 佛山科学技术学院 一种碱基编辑器及其制备方法和应用
JP2024509353A (ja) * 2021-01-25 2024-03-01 ザ・ブロード・インスティテュート・インコーポレイテッド 再プログラム可能なTnpBポリペプチド及びその使用
WO2022170044A1 (en) * 2021-02-05 2022-08-11 The General Hospital Corporation Astrocyte interleukin-3 reprograms microglia and limits alzheimer's disease
US20240141328A1 (en) * 2021-03-08 2024-05-02 Ladder Therapeutics, Inc. Assay for Massive Parallel RNA Function Perturbation Profiling
AU2022272250A1 (en) * 2021-05-14 2023-11-30 Beam Therapeutics Inc. Compositions and methods for treating transthyretin amyloidosis
EP4347818A2 (en) 2021-06-01 2024-04-10 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof
CN116751780A (zh) * 2021-06-08 2023-09-15 山东舜丰生物科技有限公司 一种具有碱基突变的同向重复序列及其应用
WO2023283622A1 (en) 2021-07-08 2023-01-12 Montana State University Crispr-based programmable rna editing
US20230167487A2 (en) 2021-07-12 2023-06-01 Labsimply, Inc. Crispr cascade assay
WO2023004391A2 (en) 2021-07-21 2023-01-26 Montana State University Nucleic acid detection using type iii crispr complex
CN113801933B (zh) * 2021-09-17 2024-03-29 上海五色石医学科技有限公司 一种人serpinb7基因突变快速分型的检测试剂盒
CN119120424A (zh) * 2021-11-02 2024-12-13 辉大(上海)生物科技有限公司 一种CRISPR-Cas12i系统
CN116254246B (zh) * 2021-12-09 2025-09-23 北京干细胞与再生医学研究院 工程化cas12b效应蛋白及其使用方法
US20230279375A1 (en) 2021-12-13 2023-09-07 Labsimply, Inc. Signal boost cascade assay
WO2023114052A1 (en) 2021-12-13 2023-06-22 Labsimply, Inc. Tuning cascade assay kinetics via molecular design
US20230295646A1 (en) * 2021-12-13 2023-09-21 Pairwise Plants Services, Inc. Model editing systems and methods relating to the same
KR102793158B1 (ko) * 2021-12-17 2025-04-07 건국대학교 산학협력단 향상된 CRISPR/Cas 시스템을 이용한 위생 지표 미생물의 검출용 키트 및 검출 방법
IL316038A (en) 2022-04-04 2024-11-01 Univ California Preparations and methods for genetic complementation
CN118995668A (zh) * 2022-04-25 2024-11-22 辉大基因治疗(新加坡)私人有限公司 新型CRISPR-Cas12i系统及其用途
WO2024015972A2 (en) * 2022-07-14 2024-01-18 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders in adult patients
CN115725743A (zh) * 2022-08-03 2023-03-03 湖南工程学院 一组检测肿瘤外泌体的探针组、试剂盒和检测体系及应用
CN115786544B (zh) * 2022-08-19 2023-11-17 湖南工程学院 一种检测牛结核分枝杆菌的试剂、试剂盒及检测方法
WO2024046307A1 (zh) * 2022-08-29 2024-03-07 北京迅识科技有限公司 一种突变的v型crispr酶及其应用
WO2024073114A1 (en) 2022-09-30 2024-04-04 Vedabio, Inc. Delivery of therapeutics in vivo via a crispr-based cascade system
US11982677B2 (en) 2022-10-02 2024-05-14 Vedabio, Inc. Dimerization screening assays
US11965205B1 (en) 2022-10-14 2024-04-23 Vedabio, Inc. Detection of nucleic acid and non-nucleic acid target molecules
CN115819543B (zh) * 2022-11-29 2023-07-21 华南师范大学 转录因子Tbx20启动子区G4调控元件在害虫防治中的应用
WO2024253707A2 (en) 2023-01-07 2024-12-12 Vedabio, Inc. Engineered nucleic acid-guided nucleases
US12060602B2 (en) 2023-01-10 2024-08-13 Vedabio, Inc. Sample splitting for multiplexed detection of nucleic acids without amplification
KR20250151430A (ko) 2023-02-15 2025-10-21 아버 바이오테크놀로지스, 인크. 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법
CN116334136A (zh) * 2023-03-30 2023-06-27 李卓 特异性清除线粒体基因组(mtDNA)的方法
WO2025064511A1 (en) * 2023-09-19 2025-03-27 Icahn School Of Medicine At Mont Sinai Bat-associated reverse transcriptase and methods of use thereof
CN120958124A (zh) * 2023-09-19 2025-11-14 广州瑞风生物科技有限公司 Cas12蛋白及其应用
WO2025067403A1 (zh) * 2023-09-28 2025-04-03 上海天泽云泰生物医药有限公司 用于治疗血红蛋白疾病的组合物和方法
CN117535354B (zh) * 2023-09-28 2024-07-19 广州瑞风生物科技有限公司 一种修复hba2基因突变的方法、组合物及其应用
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
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
CN119372179A (zh) * 2024-09-14 2025-01-28 尧唐(南京)生物科技有限公司 用于基因编辑的多肽及其应用

Family Cites Families (140)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
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
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
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4751180A (en) 1985-03-28 1988-06-14 Chiron Corporation Expression using fused genes providing for protein product
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US4935233A (en) 1985-12-02 1990-06-19 G. D. Searle And Company Covalently linked polypeptide cell modulators
DE122007000007I2 (de) 1986-04-09 2010-12-30 Genzyme Corp Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
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
WO1991006678A1 (en) 1989-10-26 1991-05-16 Sri International Dna sequencing
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
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
GB9114259D0 (en) 1991-07-02 1991-08-21 Ici Plc Plant derived enzyme and dna sequences
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
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
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
US5789156A (en) 1993-06-14 1998-08-04 Basf Ag Tetracycline-regulated transcriptional inhibitors
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
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
US5944710A (en) 1996-06-24 1999-08-31 Genetronics, Inc. Electroporation-mediated intravascular delivery
US5869326A (en) 1996-09-09 1999-02-09 Genetronics, Inc. Electroporation employing user-configured pulsing scheme
GB9907461D0 (en) 1999-03-31 1999-05-26 King S College London Neurite regeneration
GB9710049D0 (en) 1997-05-19 1997-07-09 Nycomed Imaging As Method
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
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
ATE377093T1 (de) 2000-07-07 2007-11-15 Visigen Biotechnologies Inc Sequenzbestimmung in echtzeit
GB0024550D0 (enExample) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
AU2002227156A1 (en) 2000-12-01 2002-06-11 Visigen Biotechnologies, Inc. Enzymatic nucleic acid synthesis: compositions and methods for altering monomer incorporation fidelity
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
US7057026B2 (en) 2001-12-04 2006-06-06 Solexa Limited Labelled nucleotides
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
WO2004105681A2 (en) 2003-04-28 2004-12-09 Innogenetics N.V. Cd4+ human papillomavirus (hpv) epitopes
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
MXPA06012722A (es) 2004-05-05 2007-02-14 Atugen Ag Lipidos, complejos de lipido y su uso.
WO2006069782A2 (en) 2004-12-27 2006-07-06 Silence Therapeutics Ag. Lipid complexes coated with peg and their use
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
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
AU2005274948B2 (en) 2004-07-16 2011-09-22 Genvec, Inc. Vaccines against aids comprising CMV/R-nucleic acid constructs
CA2579150C (en) 2004-09-17 2014-11-25 Pacific Biosciences Of California, Inc. Apparatus and method for analysis of molecules
GB0422877D0 (en) 2004-10-14 2004-11-17 Univ Glasgow Bioactive polymers
US7405281B2 (en) 2005-09-29 2008-07-29 Pacific Biosciences Of California, Inc. Fluorescent nucleotide analogs and uses therefor
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
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
CA2648149A1 (en) 2006-03-31 2007-11-01 Solexa, Inc. Systems and devices for sequence by synthesis analysis
JP2009534342A (ja) 2006-04-20 2009-09-24 サイレンス・セラピューティクス・アーゲー 血管内皮に特異的に送達するためのリポプレックス処方剤
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
US8343746B2 (en) 2006-10-23 2013-01-01 Pacific Biosciences Of California, Inc. Polymerase enzymes and reagents for enhanced nucleic acid sequencing
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
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
US20120164118A1 (en) 2009-05-04 2012-06-28 Fred Hutchinson Cancer Research Center Cocal vesiculovirus envelope pseudotyped retroviral vectors
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
US8927807B2 (en) 2009-09-03 2015-01-06 The Regents Of The University Of California Nitrate-responsive promoter
CA3009891C (en) 2009-12-23 2020-09-15 Novartis Ag Lipids, lipid compositions, and methods of using them
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
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
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
CA2842041A1 (en) 2012-04-18 2013-10-24 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
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
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
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
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
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
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
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
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
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
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.
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
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
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
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
CN105873568B (zh) 2013-12-05 2019-10-08 赛伦斯治疗有限公司 用于肺特异性递送的工具
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
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
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
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
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
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物
SG10201804975PA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
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
US20160304893A1 (en) 2013-12-13 2016-10-20 Cellectis Cas9 nuclease platform for microalgae genome engineering
CA2981716C (en) 2015-04-10 2022-04-12 Feldan Bio Inc. Polypeptide-based shuttle agents for improving the transduction efficiency of polypeptide cargos to the cytosol of target eukaryotic cells, uses thereof, methods and kits relating to same
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
AU2016279077A1 (en) * 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
US10648020B2 (en) * 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
WO2016205749A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
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
CN110506128A (zh) * 2016-12-09 2019-11-26 博德研究所 基于crispr效应系统的诊断
CN110612353A (zh) * 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
CN111448311B (zh) * 2017-09-09 2024-11-19 博德研究所 基于多效应子crispr的诊断系统
CN111630162A (zh) * 2017-10-04 2020-09-04 博德研究所 基于crispr效应系统的诊断
EP3727469A4 (en) * 2017-12-22 2021-12-01 The Broad Institute, Inc. NEW CRISPR ENZYMES AND SYSTEMS
WO2019126709A1 (en) * 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
EP3728576A4 (en) * 2017-12-22 2021-11-24 The Broad Institute, Inc. CAS12B SYSTEMS, METHODS AND COMPOSITIONS FOR SPECIFIC EDITING OF RNA BASES
WO2019127087A1 (zh) * 2017-12-27 2019-07-04 中国科学院动物研究所 基因组编辑系统和方法
CN109837328B (zh) * 2018-09-20 2021-07-27 中国科学院动物研究所 核酸检测方法
CN113544266A (zh) * 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
US11453907B2 (en) * 2020-03-23 2022-09-27 The Broad Institute, Inc. Crispr effector system based coronavirus diagnostics

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