CN109072235B - 通过核递送crispr/cas9追踪并操纵细胞rna - Google Patents

通过核递送crispr/cas9追踪并操纵细胞rna Download PDF

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CN109072235B
CN109072235B CN201680079817.XA CN201680079817A CN109072235B CN 109072235 B CN109072235 B CN 109072235B CN 201680079817 A CN201680079817 A CN 201680079817A CN 109072235 B CN109072235 B CN 109072235B
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rna
polypeptide
cas9
rcas9
sgrna
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CN109072235A (zh
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马克·方
兰詹·巴特拉
吉恩·杨
大卫·内勒斯
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University of California San Diego UCSD
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
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CN201680079817.XA 2015-11-23 2016-11-22 通过核递送crispr/cas9追踪并操纵细胞rna Active CN109072235B (zh)

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US201562259014P 2015-11-23 2015-11-23
US62/259,014 2015-11-23
PCT/US2016/063429 WO2017091630A1 (en) 2015-11-23 2016-11-22 Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9

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CN109072235B true CN109072235B (zh) 2023-02-28

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AU (1) AU2016359629B2 (enExample)
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Families Citing this family (73)

* 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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP4079847A1 (en) 2014-07-30 2022-10-26 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CA2990699A1 (en) 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
EP3380613B1 (en) 2015-11-23 2022-10-26 The Regents of the University of California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 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
SG11201903089RA (en) 2016-10-14 2019-05-30 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
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
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
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
AU2018283405B2 (en) 2017-06-15 2025-06-26 The Regents Of The University Of California Targeted non-viral DNA insertions
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
US10476825B2 (en) 2017-08-22 2019-11-12 Salk Institue for Biological Studies RNA targeting methods and compositions
US11970720B2 (en) 2017-08-22 2024-04-30 Salk Institute For Biological Studies RNA targeting methods and compositions
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
JP7381476B2 (ja) * 2017-10-24 2023-11-15 サンガモ セラピューティクス， インコーポレイテッド 希少疾患の処置のための方法および組成物
EP3700924A4 (en) 2017-10-27 2021-11-03 The Regents of the University of California TARGETED REPLACEMENT OF ENDOGENOUS T-CELL RECEPTORS
CN107893260B (zh) * 2017-11-27 2021-01-12 广州市锐博生物科技有限公司 高效去除核糖体rna的构建转录组测序文库的方法及试剂盒
CN108103090B (zh) * 2017-12-12 2021-06-15 中山大学附属第一医院 靶向RNA甲基化的RNA Cas9-m6A修饰载体系统及其构建方法和应用
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN108559760A (zh) * 2018-01-09 2018-09-21 陕西师范大学 基于CRISPR靶向基因组修饰技术建立荧光素酶knock-in细胞系的方法
AU2019236210B2 (en) 2018-03-14 2025-07-03 Arbor Biotechnologies, Inc. Novel CRISPR DNA targeting enzymes and systems
WO2019178428A1 (en) * 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna and rna targeting enzymes and systems
EP3810273A4 (en) * 2018-05-15 2022-03-16 The Children's Hospital of Philadelphia HTT ALLELE REMOVAL BASED ON CRISPR INTERFERENCE AND TREATMENT OF HUNTINGTON'S DISEASE
WO2019222555A1 (en) 2018-05-16 2019-11-21 Arbor Biotechnologies, Inc. Novel crispr-associated systems and components
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3575401A1 (en) * 2018-05-29 2019-12-04 Ecole Polytechnique Federale De Lausanne (Epfl) Methods of treating or preventing an aging-related disease
EP3801641A4 (en) * 2018-06-08 2022-09-28 Locanabio, Inc. Compositions and methods for the modulation of adaptive immunity
CN112930395A (zh) * 2018-06-08 2021-06-08 洛卡纳生物股份有限公司 靶向rna的融合蛋白组合物和使用方法
CN112423781A (zh) * 2018-07-13 2021-02-26 普林斯顿大学理事会 用于调节应激颗粒组装的系统和方法
WO2020069331A1 (en) * 2018-09-28 2020-04-02 The Jackson Laboratory Artificial rna-guided splicing factors
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN109706149B (zh) * 2018-11-29 2020-12-18 华中农业大学 一种改进的16S-seq方法及其应用
RU2694396C1 (ru) * 2018-12-14 2019-07-12 Федеральное Бюджетное Учреждение Науки "Центральный Научно-Исследовательский Институт Эпидемиологии" Федеральной Службы По Надзору В Сфере Защиты Прав Потребителей И Благополучия Человека РНК-проводник St10 для использования в высокоспецифической системе нуклеаз Streptococcus thermophilus CRISPR/Cas9 (StCas9) и применение указанного РНК-проводника и белка StCas9 для подавления экспрессии вируса гепатита B в клетке-хозяине и для элиминации вирусной ДНК из клетки-хозяина
AU2020208346A1 (en) * 2019-01-14 2021-07-29 University Of Rochester Targeted nuclear RNA cleavage and polyadenylation with CRISPR-cas
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020160125A1 (en) * 2019-01-29 2020-08-06 Flagship Pioneering Innovations V, Inc. Compositions comprising an endonuclease and methods for purifying an endonuclease
LU101118B1 (en) 2019-02-06 2020-08-06 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Method for detecting a specific splice event of a gene of interest
CN109811072B (zh) * 2019-02-28 2022-05-06 广州微远医疗器械有限公司 用于结核分枝杆菌复合群的crispr检测引物组及其用途
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
CN110004181B (zh) * 2019-03-20 2022-10-11 东北农业大学 一种附加型CRISPR/Cas9表达载体及其构建方法与应用
WO2020198640A1 (en) * 2019-03-27 2020-10-01 Massachusetts Institute Of Technology Programmable imaging methods and compositions
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12139517B2 (en) 2019-04-23 2024-11-12 Sangamo Therapeutics, Inc. Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
CN110714008A (zh) * 2019-10-17 2020-01-21 南通大学 核苷酸序列构建的crispr重组质粒靶向编辑iss序列的方法
CA3163565A1 (en) * 2019-12-02 2021-06-10 Council Of Scientific & Industrial Research Method and kit for detection of polynucleotide
GB2614813B (en) 2020-05-08 2025-05-07 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
US12139710B2 (en) 2020-10-28 2024-11-12 The Regents Of The University Of California Methods of modulating RNA translation
GB202105455D0 (en) 2021-04-16 2021-06-02 Ucl Business Ltd Composition
EP4095243A1 (en) * 2021-05-25 2022-11-30 European Molecular Biology Laboratory System for hybridization-based precision genome cleavage and editing, and uses thereof
WO2023023584A2 (en) 2021-08-19 2023-02-23 Eclipse Bioinnovations, Inc. Methods for detecting rna binding protein complexes
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
CR20240195A (es) 2021-10-14 2024-06-20 Arsenal Biosciences Inc Células inmunitarias que tienen arnhc coexpresados y sistemas de compuerta lógica
WO2023129973A2 (en) * 2021-12-30 2023-07-06 Eclipse Bioinnovations, Inc. Methods for detecting rna modification targets on a gene
WO2023205637A1 (en) * 2022-04-18 2023-10-26 Locanabio, Inc. Rna-targeting compositions and methods for treating c9/orf72 diseases
CN115044620B (zh) * 2022-06-30 2024-01-05 上海海洋大学 微卫星重复序列敲除突变体的构建方法和应用
CN115927425B (zh) * 2022-07-14 2025-03-25 华中农业大学 一种嗜热栖热菌基因编辑质粒及在生产超氧化物歧化酶中的应用
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
CN117925787A (zh) * 2024-01-17 2024-04-26 北京医院 基于“内-外”双重信号放大的DCasFISH染色体多色原位成像系统

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015089351A1 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015089277A1 (en) * 2013-12-12 2015-06-18 The Regents Of The University Of California Methods and compositions for modifying a single stranded target nucleic acid

Family Cites Families (90)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
NZ239247A (en) 1990-08-03 1993-11-25 Sterling Drug Inc Oligonucleosides containing a non-phosphate inter nucleoside linkage
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
WO1992005186A1 (en) 1990-09-20 1992-04-02 Gilead Sciences Modified internucleoside linkages
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
DE69407032T2 (de) 1993-03-31 1998-07-02 Sanofi Sa Oligonucleotide mit amidverkettungen die phosphoesterverkettungen einsetzen
DE19502912A1 (de) 1995-01-31 1996-08-01 Hoechst Ag G-Cap Stabilisierte Oligonucleotide
US6042820A (en) 1996-12-20 2000-03-28 Connaught Laboratories Limited Biodegradable copolymer containing α-hydroxy acid and α-amino acid units
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
ATE293123T1 (de) 1997-09-12 2005-04-15 Exiqon As Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
DE69940196D1 (de) 1998-04-15 2009-02-12 Hutchinson Fred Cancer Res Methoden und vektorenkonstrukte zur erzeugung von transgene nagetieren, welche ein heterologes gen ubiquitär exprimieren
US6472375B1 (en) 1998-04-16 2002-10-29 John Wayne Cancer Institute DNA vaccine and methods for its use
US7078387B1 (en) 1998-12-28 2006-07-18 Arch Development Corp. Efficient and stable in vivo gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
WO2001048190A2 (en) 1999-12-23 2001-07-05 Exiqon A/S Therapeutic uses of lna-modified oligonucleotides
WO2001075097A2 (en) 2000-03-31 2001-10-11 Cambridge Antibody Technology Limited Improvements to ribosome display
US8592569B2 (en) 2007-11-22 2013-11-26 Japan Science And Technology Agency Small RNA-dependent translational regulatory system in cell or artificial cell model
US9580714B2 (en) 2010-11-24 2017-02-28 The University Of Western Australia Peptides for the specific binding of RNA targets
CN106626106A (zh) 2011-01-07 2017-05-10 坂东机工株式会社 用于刻划碳化硅板的方法和设备
JP6164488B2 (ja) 2011-10-21 2017-07-19 国立大学法人九州大学 Ｐｐｒモチーフを利用したｒｎａ結合性蛋白質の設計方法及びその利用
EP3858847A1 (en) * 2011-11-30 2021-08-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
WO2013130683A2 (en) 2012-02-27 2013-09-06 Amunix Operating Inc. Xten conjugate compositions and methods of making same
CN105209621B (zh) * 2012-12-12 2021-05-25 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
AU2013359199C1 (en) 2012-12-12 2021-06-17 Massachusetts Institute Of Technology 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
AU2014207618A1 (en) * 2013-01-16 2015-08-06 Emory University Cas9-nucleic acid complexes and uses related thereto
TWI484033B (zh) 2013-01-25 2015-05-11 Univ China Medical 培養幹細胞之方法及套組
WO2014186585A2 (en) * 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
US11685935B2 (en) * 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
JP7065564B2 (ja) * 2013-05-29 2022-05-12 セレクティス Ｃａｓ９ニッカーゼ活性を用いて正確なｄｎａ切断をもたらすための方法
CN105517579B (zh) * 2013-07-10 2019-11-15 哈佛大学校长及研究员协会 用于RNA向导的基因调节和编辑的正交Cas9蛋白
MX2016002044A (es) 2013-08-16 2016-08-17 Rana Therapeutics Inc Composiciones y metodos para modular el acido ribonucleico.
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US10822606B2 (en) * 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
CN106459957B (zh) 2014-03-05 2020-03-20 国立大学法人神户大学 用于特异性转变靶向dna序列的核酸碱基的基因组序列的修饰方法、及其使用的分子复合体
CN105338513B (zh) 2014-08-08 2019-12-10 中兴通讯股份有限公司 设备到设备业务处理方法及装置
RU2711506C2 (ru) 2014-12-17 2020-01-17 ПРОКЬЮЭР ТЕРАПЬЮТИКС II Би.Ви. Редактирование целевой рнк
WO2016106236A1 (en) * 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
US10330674B2 (en) 2015-01-13 2019-06-25 Massachusetts Institute Of Technology Pumilio domain-based modular protein architecture for RNA binding
US10920221B2 (en) 2015-05-13 2021-02-16 President And Fellows Of Harvard College Methods of making and using guide RNA for use with Cas9 systems
WO2016191684A1 (en) 2015-05-28 2016-12-01 Finer Mitchell H Genome editing vectors
EP3303634B1 (en) 2015-06-03 2023-08-30 The Regents of The University of California Cas9 variants and methods of use thereof
EP3334823B1 (en) 2015-06-05 2024-05-22 The Regents of The University of California Method and kit for generating crispr/cas guide rnas
US20160362667A1 (en) 2015-06-10 2016-12-15 Caribou Biosciences, Inc. CRISPR-Cas Compositions and Methods
WO2016201138A1 (en) 2015-06-12 2016-12-15 The Regents Of The University Of California Reporter cas9 variants and methods of use thereof
EP3323890A4 (en) 2015-07-14 2019-01-30 Fukuoka University METHOD OF INDUSTRYING SITE-SPECIFIC RNA MUTATIONS, TARGETED EDITING GUIDE RNA USED IN THE METHOD AND TARGET RNA TARGET EDITING GUIDE RNA COMPLEX
PT4140491T (pt) 2015-09-21 2023-12-05 Trilink Biotechnologies Llc Métolo para a sintetização de arn capeados em 5
WO2017053312A1 (en) 2015-09-21 2017-03-30 The Regents Of The University Of California Compositions and methods for target nucleic acid modification
EP3380613B1 (en) * 2015-11-23 2022-10-26 The Regents of the University of California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
AU2017283713B2 (en) 2016-06-17 2021-04-08 Massachusetts Institute Of Technology Type VI CRISPR orthologs and systems
LT6525B (lt) 2016-06-29 2018-05-10 Uab Pixpro Skaitmeninių vaizdų skyros didinimo būdas taikant unikalų dalinai persidengiančių mažos skyros vaizdų apdorojimą
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
CN119351474A (zh) 2017-02-22 2025-01-24 克里斯珀医疗股份公司 用于基因编辑的组合物和方法
WO2018183703A1 (en) 2017-03-31 2018-10-04 NeuroDiagnostics LLC Lymphocyte-based morphometric test for alzheimer's disease
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
US10476825B2 (en) 2017-08-22 2019-11-12 Salk Institue for Biological Studies RNA targeting methods and compositions
JP2020535802A (ja) 2017-09-21 2020-12-10 ザ・ブロード・インスティテュート・インコーポレイテッド 標的化核酸編集のための系、方法、及び組成物
CN108103090B (zh) 2017-12-12 2021-06-15 中山大学附属第一医院 靶向RNA甲基化的RNA Cas9-m6A修饰载体系统及其构建方法和应用
EP3728588A4 (en) 2017-12-22 2022-03-09 The Broad Institute, Inc. CAS12A SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED EDIT OF RNA BASES
CN112513250A (zh) 2018-04-20 2021-03-16 加利福尼亚大学董事会 用于跟踪和操纵细胞rna的融合蛋白和融合核糖核酸
US20210332344A1 (en) 2018-08-31 2021-10-28 The Regents Of The University Of California Directed modification of rna
WO2020047489A1 (en) 2018-08-31 2020-03-05 The Regents Of The University Of California Directed pseudouridylation of rna
CN110055284A (zh) 2019-04-15 2019-07-26 中山大学 一种基于PspCas13b-Alkbh5单基因特异性m6A修饰编辑方法
WO2020214806A1 (en) 2019-04-16 2020-10-22 The Regents Of The University Of California Protein translational control

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015089351A1 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015089277A1 (en) * 2013-12-12 2015-06-18 The Regents Of The University Of California Methods and compositions for modifying a single stranded target nucleic acid

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotesn;Luke A. Gilbert等;《Cell》;20130718;第442-451页 *
Programmable RNA recognition and cleavage byCRISPR/Cas9;Mitchell R O‘Connell等;《nature》;20140928(第516期);摘要及第264页第3段及图2 *

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