PT3380613T - Deteção e manipulação de arn celular através da administração nuclear de crispr/cas9 - Google Patents

Deteção e manipulação de arn celular através da administração nuclear de crispr/cas9

Info

Publication number
PT3380613T
PT3380613T PT168227668T PT16822766T PT3380613T PT 3380613 T PT3380613 T PT 3380613T PT 168227668 T PT168227668 T PT 168227668T PT 16822766 T PT16822766 T PT 16822766T PT 3380613 T PT3380613 T PT 3380613T
Authority
PT
Portugal
Prior art keywords
cas9
crispr
tracking
cellular rna
rna via
Prior art date
Application number
PT168227668T
Other languages
English (en)
Original Assignee
Univ California
Fang Mark
Batra Ranjan
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ California, Fang Mark, Batra Ranjan filed Critical Univ California
Publication of PT3380613T publication Critical patent/PT3380613T/pt

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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 [CRISPRs]
    • 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
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurosurgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
PT168227668T 2015-11-23 2016-11-22 Deteção e manipulação de arn celular através da administração nuclear de crispr/cas9 PT3380613T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562259014P 2015-11-23 2015-11-23

Publications (1)

Publication Number Publication Date
PT3380613T true PT3380613T (pt) 2022-12-02

Family

ID=57750540

Family Applications (1)

Application Number Title Priority Date Filing Date
PT168227668T PT3380613T (pt) 2015-11-23 2016-11-22 Deteção e manipulação de arn celular através da administração nuclear de crispr/cas9

Country Status (10)

Country Link
US (4) US20170145394A1 (pt)
EP (2) EP4163374A1 (pt)
JP (2) JP2019500899A (pt)
CN (1) CN109072235B (pt)
AU (1) AU2016359629B2 (pt)
CA (1) CA3005968A1 (pt)
ES (1) ES2930643T3 (pt)
PT (1) PT3380613T (pt)
SG (2) SG10202004557QA (pt)
WO (1) WO2017091630A1 (pt)

Families Citing this family (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
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
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
JP2018519811A (ja) 2015-06-29 2018-07-26 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 修飾ｃｒｉｓｐｒ ｒｎａ及び修飾単一ｃｒｉｓｐｒ ｒｎａならびにその使用
CN108699116A (zh) 2015-10-23 2018-10-23 哈佛大学的校长及成员们 用于基因编辑的演化的cas9蛋白
PT3380613T (pt) 2015-11-23 2022-12-02 Univ California Deteção e manipulação de arn celular através da administração nuclear de crispr/cas9
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
EP3497214B1 (en) 2016-08-09 2023-06-28 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
CA3039928A1 (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
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592777A1 (en) 2017-03-10 2020-01-15 President and Fellows of Harvard College Cytosine to guanine base editor
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
CA3062595A1 (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
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
KR20240027153A (ko) 2017-06-15 2024-02-29 더 리전트 오브 더 유니버시티 오브 캘리포니아 표적화된 비-바이러스 dna 삽입
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
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
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
AU2018355343A1 (en) * 2017-10-24 2020-05-07 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of rare diseases
IL274179B2 (en) 2017-10-27 2024-02-01 Univ 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
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
FI3765616T3 (fi) * 2018-03-14 2023-08-29 Arbor Biotechnologies Inc Uusia CRISPR-DNA- ja RNA-kohdennusentsyymejä ja -järjestelmiä
CN112041444A (zh) 2018-03-14 2020-12-04 阿伯生物技术公司 新型crispr dna靶向酶及系统
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
EP3575401A1 (en) * 2018-05-29 2019-12-04 Ecole Polytechnique Federale De Lausanne (Epfl) Methods of treating or preventing an aging-related disease
US20190382759A1 (en) * 2018-06-08 2019-12-19 Locana, 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
EP3820494A4 (en) * 2018-07-13 2022-07-13 The Trustees of Princeton University SYSTEM AND METHOD FOR MODULATING THE ASSEMBLY OF STRESS PELLETS
EP3856898A4 (en) * 2018-09-28 2022-06-22 The Jackson Laboratory ARTIFICIAL RNA-GUIDED SPLICING FACTORS
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
US20220090088A1 (en) * 2019-01-14 2022-03-24 University Of Rochester Targeted Nuclear RNA Cleavage and Polyadenylation with CRISPR-Cas
US20220249625A1 (en) * 2019-01-29 2022-08-11 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
WO2020191243A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
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
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
AU2020394910A1 (en) * 2019-12-02 2022-06-23 Council Of Scientific & Industrial Research Method and kit for detection of polynucleotide
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
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
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
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

Family Cites Families (92)

* 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
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
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
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
JPH06502300A (ja) 1990-08-03 1994-03-17 サノフィ 遺伝子発現の抑制のための化合物及び方法
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
AU662298B2 (en) 1990-09-20 1995-08-31 Gilead Sciences, Inc. Modified internucleoside linkages
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
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
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
CA2159629A1 (en) 1993-03-31 1994-10-13 Sanofi Oligonucleotides with amide linkages replacing phosphodiester linkages
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
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
WO1999053017A2 (en) 1998-04-15 1999-10-21 Fred Hutchinson Cancer Research Center Methods and vector constructs for making transgenic non-human animals which ubiquitously express a heterologous gene
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
DK1178999T3 (da) 1999-05-04 2007-08-06 Santaris Pharma As L-RIBO-LNA-analoger
JP2003524637A (ja) 1999-12-23 2003-08-19 エクシコン エ／エス Ｌｎａ修飾オリゴヌクレオチドの治療上の使用
DK1268766T3 (da) 2000-03-31 2006-04-10 Cambridge Antibody Tech Forbedringer af ribosom-display
EP2221371B1 (en) 2007-11-22 2013-11-13 Japan Science and Technology Agency Translation regulation system in cell or artificial cell model by using low-molecular-weight rna
WO2012068627A1 (en) 2010-11-24 2012-05-31 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
ES2751126T3 (es) 2011-10-21 2020-03-30 Univ Kyushu Nat Univ Corp Método de diseño para proteína de unión a ARN usando motivo de PPR, y uso del mismo
EP3858847A1 (en) 2011-11-30 2021-08-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
CN107496932A (zh) 2012-02-27 2017-12-22 阿穆尼克斯运营公司 Xten缀合组合物和制造其的方法
SG10201801969TA (en) * 2012-12-12 2018-04-27 Broad Inst Inc Engineering and Optimization of Improved Systems, Methods and Enzyme Compositions for Sequence Manipulation
CN105164264B (zh) 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵和治疗应用的系统、方法和组合物的递送、工程化和优化
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CA2898184A1 (en) * 2013-01-16 2014-07-24 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
EP3730615A3 (en) * 2013-05-15 2020-12-09 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
CA2913869C (en) * 2013-05-29 2023-01-24 Cellectis New compact scaffold of cas9 in the type ii crispr system
EP3004349B1 (en) * 2013-05-29 2018-03-28 Cellectis S.A. A method for producing precise dna cleavage using cas9 nickase activity
EP3019204B1 (en) * 2013-07-10 2020-01-01 President and Fellows of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
AU2014306416B2 (en) 2013-08-16 2021-02-25 Translate Bio Ma, Inc. Compositions and methods for modulating RNA
US9388430B2 (en) * 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
WO2015048690A1 (en) * 2013-09-27 2015-04-02 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
EP3835419A1 (en) 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
CA2932472A1 (en) 2013-12-12 2015-06-18 Massachusetts Institute Of Technology Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015133554A1 (ja) 2014-03-05 2015-09-11 国立大学法人神戸大学 標的化したｄｎａ配列の核酸塩基を特異的に変換するゲノム配列の改変方法及びそれに用いる分子複合体
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
CN107109413B (zh) 2014-12-17 2021-03-09 ProQR治疗上市公司Ⅱ 靶向的rna编辑
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
WO2016183402A2 (en) 2015-05-13 2016-11-17 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
WO2016196655A1 (en) 2015-06-03 2016-12-08 The Regents Of The University Of California Cas9 variants and methods of use thereof
US11279926B2 (en) 2015-06-05 2022-03-22 The Regents Of The University Of California Methods and compositions for generating CRISPR/Cas guide RNAs
US20160362667A1 (en) 2015-06-10 2016-12-15 Caribou Biosciences, Inc. CRISPR-Cas Compositions and Methods
US20180142222A1 (en) 2015-06-12 2018-05-24 The Regents Of The University Of California Reporter cas9 variants and methods of use thereof
WO2017010556A1 (ja) 2015-07-14 2017-01-19 学校法人福岡大学 部位特異的ｒｎａ変異導入方法およびそれに使用する標的編集ガイドｒｎａならびに標的ｒｎａ－標的編集ガイドｒｎａ複合体
EP3906789B1 (en) 2015-09-21 2023-11-22 TriLink BioTechnologies, LLC Initiating capped oligonucleotide primers for synthesizing 5'-capped rnas
WO2017053312A1 (en) 2015-09-21 2017-03-30 The Regents Of The University Of California Compositions and methods for target nucleic acid modification
PT3380613T (pt) * 2015-11-23 2022-12-02 Univ California Deteção e manipulação de arn celular através da administração nuclear de crispr/cas9
WO2017219027A1 (en) 2016-06-17 2017-12-21 The Broad Institute Inc. 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
US11920148B2 (en) 2017-02-22 2024-03-05 Crispr Therapeutics Ag Compositions and methods for gene editing
KR102357045B1 (ko) 2017-03-31 2022-01-28 뉴로디아그노스틱스 엘엘씨 알츠하이머 질환에 대한 림프구-기반 형태계측 시험
CA3062595A1 (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
US11168322B2 (en) 2017-06-30 2021-11-09 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 RNA BASE EDITING
AU2019255798A1 (en) 2018-04-20 2020-11-26 The Regents Of The University Of California Fusion proteins and fusion ribonucleic acids for tracking and manipulating cellular RNA
US20210340197A1 (en) 2018-08-31 2021-11-04 The Regents Of The University Of California Directed pseudouridylation of rna
US20210332344A1 (en) 2018-08-31 2021-10-28 The Regents Of The University Of California Directed modification 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

Also Published As

Publication number Publication date
WO2017091630A1 (en) 2017-06-01
US20200239863A1 (en) 2020-07-30
US20230365951A1 (en) 2023-11-16
EP3380613A1 (en) 2018-10-03
SG11201804372PA (en) 2018-06-28
CA3005968A1 (en) 2017-06-01
US20190040370A1 (en) 2019-02-07
US20170145394A1 (en) 2017-05-25
AU2016359629B2 (en) 2023-03-09
JP2022081503A (ja) 2022-05-31
CN109072235B (zh) 2023-02-28
ES2930643T3 (es) 2022-12-20
AU2016359629A1 (en) 2018-06-07
CN109072235A (zh) 2018-12-21
SG10202004557QA (en) 2020-06-29
EP3380613B1 (en) 2022-10-26
WO2017091630A4 (en) 2017-08-24
EP4163374A1 (en) 2023-04-12
US11667903B2 (en) 2023-06-06
JP2019500899A (ja) 2019-01-17

Similar Documents

Publication Publication Date Title
SG10202004557QA (en) Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
EP3622062A4 (en) DIRECTED EDITING OF CELLULAR RNA BY NUCLEAR ADMINISTRATION OF CRISPR / CAS9
IL290678B1 (en) New crispr enzymes and systems
HK1258288A1 (zh) 核酸酶非依賴性靶向基因編輯平台及其用途
SG11202001208XA (en) Rna targeting methods and compositions
EP3374494A4 (en) CRISPR COMPOSITIONS AND METHODS OF USE FOR GENE THERAPY
IL259100B1 (en) Therapeutic targets for human dystrophin gene repair through gene editing and methods for use
HK1253674A1 (zh) 用於具有增强的穩定性的瞬時基因療法的組合物和方法
EP3307762A4 (en) CAS9 REPORTER VARIANTS AND METHODS OF USE
EP3313989A4 (en) Modified crispr rna and modified single crispr rna and uses thereof
EP3359644A4 (en) NOVEL RNA-GUIDED NUCLEASES AND USES THEREOF
EP3397757A4 (en) NEW TRANSPOSASES ASSOCIATED WITH CRISPR AND USES THEREOF
EP3303634A4 (en) CASE VARIANTS9 AND METHODS OF USE THEREOF
EP3362104A4 (en) METHODS AND COMPOSITIONS USING CPF1 FOR RNA-GUIDED GENETIC EDITION
IL250744A0 (en) A method for preparing flavociclib and its pharmaceutical preparations
EP3245232A4 (en) Heterodimeric cas9 and methods of use thereof
GB201510296D0 (en) Thermostable CAS9 nucleases
HK1254608A1 (zh) 改良的地拉羅司製劑及其製備方法
ZA201900225B (en) Methods and compositions for gene expression in plants
ZA202104969B (en) Compositions comprising 15-hepe and methods of using the same
GB201519128D0 (en) Solid forms and methods of preparing the same
HK1252778A1 (zh) 細胞擴增方法和治療組合物
IL255178B (en) Strigolactones or their derivatives for use in increasing the yield of a cultivated plant
PL3242957T3 (pl) Kompozycje zawierające bakterie i sposoby ich wykorzystania do leczenia i/lub zapobiegania chorobom żołądkowo-jelitowym, metabolicznym i/lub innym
HK1247136A1 (zh) 包含15-hepe的組合物和其使用方法