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)
Portuguese (pt)
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

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    • 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 [RNase]; Deoxyribonucleases [DNase]
    • 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 [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
    • 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

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  • 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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (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

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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 (enExample)
EP (2) EP3380613B1 (enExample)
JP (2) JP2019500899A (enExample)
CN (1) CN109072235B (enExample)
AU (1) AU2016359629B2 (enExample)
CA (1) CA3005968A1 (enExample)
ES (1) ES2930643T3 (enExample)
PT (1) PT3380613T (enExample)
SG (2) SG11201804372PA (enExample)
WO (1) WO2017091630A1 (enExample)

Families Citing this family (76)

* 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
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 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
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
AU2016359629B2 (en) * 2015-11-23 2023-03-09 Ranjan BATRA 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
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018119359A1 (en) 2016-12-23 2018-06-28 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
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
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
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include 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
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
MY200418A (en) 2017-06-15 2023-12-25 Univ California Targeted non-viral dna insertions
EP3658573A1 (en) 2017-07-28 2020-06-03 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
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
EP3716767A4 (en) * 2017-10-24 2021-11-24 Sangamo Therapeutics, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF RARE DISEASES
SG11202003798TA (en) 2017-10-27 2020-05-28 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
US12406749B2 (en) 2017-12-15 2025-09-02 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
PL3765615T3 (pl) 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
DK3765616T3 (da) * 2018-03-14 2023-08-21 Arbor Biotechnologies Inc Nye enzymer og systemer målrettet crispr dna og rna
WO2019222437A1 (en) * 2018-05-15 2019-11-21 The Children's Hospital Of Philadelphia Crispr interference based htt allelic suppression and treatment of huntington 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
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
JP2021526860A (ja) * 2018-06-08 2021-10-11 ロックアネイビオ， インコーポレイテッド 適応免疫をモジュレートするための組成物および方法
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
EP3820494A4 (en) * 2018-07-13 2022-07-13 The Trustees of Princeton University SYSTEM AND METHOD FOR MODULATION OF A STRESS GRANULE ASSEMBLY
US20210388351A1 (en) * 2018-09-28 2021-12-16 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
CN113544267B (zh) * 2019-01-14 2025-04-18 罗切斯特大学 使用CRISPR-Cas进行靶向核RNA裂解和聚腺苷酸化
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
WO2020191241A1 (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
US12637682B2 (en) 2019-04-16 2026-05-26 The Regents Of The University Of California Protein translational control
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
JP7618581B2 (ja) 2019-04-23 2025-01-21 サンガモ セラピューティクス， インコーポレイテッド ９番染色体オープンリーディングフレーム７２遺伝子発現のモジュレーターおよびその使用
US12435330B2 (en) 2019-10-10 2025-10-07 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
EP4073262A4 (en) * 2019-12-02 2023-12-27 Council of Scientific & Industrial Research METHOD AND KIT FOR DETECTING POLYNUCLEOTIDE
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu 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
JP2024533163A (ja) 2021-09-03 2024-09-12 タシト・セラピューティクス・インコーポレーテッド スプライソソーム構成要素のリクルートを介したｒｎａ編集
MX2024003887A (es) 2021-10-14 2024-07-09 Arsenal Biosciences Inc Células inmunitarias que tienen arnch coespresados 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
JP2026523341A (ja) 2023-05-15 2026-07-14 エヌクロマ・バイオ，インコーポレーテッド Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法
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
CN121380070B (zh) * 2025-11-10 2026-04-10 上海交通大学医学院附属仁济医院 靶向sfpq的腺相关病毒及其在制备治疗神经母细胞瘤的药物中的用途

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
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
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
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
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
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
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
HU9501974D0 (en) 1993-03-31 1995-09-28 Sterling Winthrop Inc 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
EP1015469B2 (en) 1997-09-12 2015-11-18 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
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
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
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
WO2013082548A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
CN104302408B (zh) 2012-02-27 2016-12-14 阿穆尼克斯运营公司 Xten缀合组合物和制造其的方法
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
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst 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
WO2014113493A1 (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
EP2997146A4 (en) * 2013-05-15 2017-04-26 Sangamo BioSciences, Inc. Methods and compositions for treatment of a genetic condition
EP3004339B1 (en) * 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
DK3004349T3 (en) * 2013-05-29 2018-06-06 Cellectis Sa A method for producing precise DNA cleavage using CAS9 nickase activity
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
CA2921556A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating rna
US9322037B2 (en) * 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI 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
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
KR20160097338A (ko) * 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도
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
EP3613854A1 (en) 2014-03-05 2020-02-26 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
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
AU2015367378A1 (en) 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
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
EP3302575A4 (en) 2015-05-28 2019-01-16 Coda Biotherapeutics GEMONEDITIERUNGSVEKTOREN
US10392607B2 (en) 2015-06-03 2019-08-27 The Regents Of The University Of California Cas9 variants and methods of use thereof
CA3001683C (en) 2015-06-05 2024-06-04 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
WO2016201138A1 (en) 2015-06-12 2016-12-15 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
US20180237800A1 (en) 2015-09-21 2018-08-23 The Regents Of The University Of California Compositions and methods for target nucleic acid modification
DK4276189T3 (da) 2015-09-21 2026-05-26 Trilink Biotechnologies Llc Initierende oligonukleotidprimere med kappe til syntesering af 5'-ende RNA'er med kappe
AU2016359629B2 (en) 2015-11-23 2023-03-09 Ranjan BATRA 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
AU2018224387B2 (en) 2017-02-22 2024-08-08 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
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
KR102338449B1 (ko) 2017-09-21 2021-12-10 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
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
WO2019126762A2 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
CA3097857A1 (en) 2018-04-20 2019-10-24 The Regents Of The University Of California Fusion proteins and fusion ribonucleic acids for tracking and manipulating cellular rna
US20210332344A1 (en) 2018-08-31 2021-10-28 The Regents Of The University Of California Directed modification of rna
US20210340197A1 (en) 2018-08-31 2021-11-04 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
US12637682B2 (en) 2019-04-16 2026-05-26 The Regents Of The University Of California Protein translational control

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