MA47239A - Stratégie optimisée pour des modifications par saut d'exon à l'aide de crispr/cas9 avec des séquences de guidage triple - Google Patents

Stratégie optimisée pour des modifications par saut d'exon à l'aide de crispr/cas9 avec des séquences de guidage triple

Info

Publication number
MA47239A
MA47239A MA047239A MA47239A MA47239A MA 47239 A MA47239 A MA 47239A MA 047239 A MA047239 A MA 047239A MA 47239 A MA47239 A MA 47239A MA 47239 A MA47239 A MA 47239A
Authority
MA
Morocco
Prior art keywords
cas9
crispr
exon skip
optimized strategy
triple
Prior art date
Application number
MA047239A
Other languages
English (en)
Inventor
Leonela Amoasii
Eric Olson
Original Assignee
Univ Texas
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 Texas filed Critical Univ Texas
Publication of MA47239A publication Critical patent/MA47239A/fr

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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • 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
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart cells
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0658—Skeletal muscle cells, e.g. myocytes, myotubes, myoblasts
    • C12N5/0659—Satellite cells
    • C—CHEMISTRY; METALLURGY
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/066—Tenocytes; Tendons, Ligaments
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0661—Smooth muscle cells
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
    • C—CHEMISTRY; METALLURGY
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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 [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/90—Vectors containing a transposable element

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  • Organic Chemistry (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Rheumatology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Transplantation (AREA)
  • Epidemiology (AREA)
MA047239A 2017-01-05 2018-01-05 Stratégie optimisée pour des modifications par saut d'exon à l'aide de crispr/cas9 avec des séquences de guidage triple MA47239A (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762442606P 2017-01-05 2017-01-05
US201762544449P 2017-08-11 2017-08-11
US201762596298P 2017-12-08 2017-12-08

Publications (1)

Publication Number Publication Date
MA47239A true MA47239A (fr) 2019-11-13

Family

ID=61193018

Family Applications (1)

Application Number Title Priority Date Filing Date
MA047239A MA47239A (fr) 2017-01-05 2018-01-05 Stratégie optimisée pour des modifications par saut d'exon à l'aide de crispr/cas9 avec des séquences de guidage triple

Country Status (21)

Country Link
US (1) US20190338311A1 (fr)
EP (1) EP3565897A1 (fr)
JP (1) JP2020503869A (fr)
KR (1) KR102606174B1 (fr)
CN (1) CN110506115A (fr)
AU (1) AU2018205521A1 (fr)
BR (1) BR112019013962A2 (fr)
CA (1) CA3048635A1 (fr)
CL (1) CL2019001882A1 (fr)
CO (1) CO2019008181A2 (fr)
CR (1) CR20190326A (fr)
DO (1) DOP2019000179A (fr)
EC (1) ECSP19056408A (fr)
IL (1) IL267786A (fr)
JO (1) JOP20190166A1 (fr)
MA (1) MA47239A (fr)
MX (1) MX2019008064A (fr)
PE (1) PE20191357A1 (fr)
PH (1) PH12019501561A1 (fr)
SG (1) SG11201906147VA (fr)
WO (1) WO2018129296A1 (fr)

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WO2013163628A2 (fr) 2012-04-27 2013-10-31 Duke University Correction génétique de gènes ayant subi une mutation
EP3256487A4 (fr) 2015-02-09 2018-07-18 Duke University Compositions et procédés pour l'édition de l'épigénome
EP4089175A1 (fr) 2015-10-13 2022-11-16 Duke University Ingénierie génomique avec systèmes crispr de type i dans des cellules eucaryotes
JPWO2018179578A1 (ja) * 2017-03-30 2020-02-06 国立大学法人京都大学 ゲノム編集によるエクソンスキッピング誘導方法
EP3735462A1 (fr) * 2018-01-05 2020-11-11 The Board of Regents of The University of Texas System Compositions crispr/cas9 thérapeutiques et méthodes d'utilisation
WO2019246480A1 (fr) * 2018-06-21 2019-12-26 The Board Of Regents Of The University Of Texas System Correction des délétions de l'exon 43, de l'exon 45 ou de l'exon 52 de la dystrophine dans la dystrophie musculaire de duchenne
CA3108289A1 (fr) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de la dystrophie musculaire facio-scapulo-humerale
MX2021001281A (es) 2018-08-02 2021-07-15 Dyne Therapeutics Inc Complejos dirigidos a músculos y sus usos para el tratamiento de distrofinopatías.
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20220017888A1 (en) * 2018-12-11 2022-01-20 Kyoto University Method for inducing deletion in genomic dna
WO2020142714A1 (fr) * 2019-01-04 2020-07-09 Exonics Therapeutics, Inc. Cassette d'expression d'aav et vecteurs aav la comprenant
CN113795587A (zh) * 2019-03-07 2021-12-14 纽约市哥伦比亚大学理事会 使用Tn7样转座子进行RNA引导的DNA整合
WO2020210214A1 (fr) * 2019-04-12 2020-10-15 The Regents Of The University Of California Compositions et méthodes pour modifier des gènes de la dystrophine
JP2022526669A (ja) * 2019-04-12 2022-05-25 デューク ユニバーシティ ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物
WO2020225606A1 (fr) 2019-05-08 2020-11-12 Crispr Therapeutics Ag Systèmes de vecteurs crispr/cas en deux parties pour le traitement de dmd
KR102264115B1 (ko) * 2019-05-10 2021-06-14 한국과학기술연구원 무-운반체 다중 CRISPR/Cas 9 유전자 편집 복합체 및 그의 용도
CN110499333A (zh) * 2019-08-01 2019-11-26 广州德赫生物科技有限公司 用于修复dmd基因突变的核酸序列及系统
WO2021072276A1 (fr) * 2019-10-11 2021-04-15 Yale University Compositions et procédés de régulation à la hausse d'isoformes de la dystrophine comme thérapie contre la dystrophie musculaire de duchenne (dmd)
CN114846146B (zh) * 2019-10-29 2024-04-12 基恩科雷有限责任公司 用于增加CRISPR/Cas12f1系统的效率的工程化引导RNA及其用途
CN111172191B (zh) * 2020-02-21 2020-12-22 浙江大学 一种高效基因敲除载体及其应用
CN115011598A (zh) * 2020-09-02 2022-09-06 西湖大学 杜氏肌营养不良症相关的外显子剪接增强子、sgRNA、基因编辑工具及应用
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2023159103A1 (fr) * 2022-02-17 2023-08-24 The Board Of Regents Of The University Of Texas System Variant crispr/spcas9 et procédés pour une correction améliorée de mutations de dystrophie musculaire de duchenne
WO2023172115A1 (fr) * 2022-03-10 2023-09-14 주식회사 진코어 Compositions et procédés pour augmenter l'efficacité de délétion d'un segment d'acide nucléique par modulation de la voie de réparation nhej
CN115820642B (zh) * 2022-11-11 2023-10-10 昆明理工大学 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统

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DK1737966T3 (da) * 2004-04-02 2012-07-09 Univ Texas Cancerspecifik promotorer
DE202013012241U1 (de) 2012-05-25 2016-01-18 Emmanuelle Charpentier Zusammensetzungen für die durch RNA gesteuerte Modifikation einer Ziel-DNA und für die durch RNA gesteuerte Modulation der Transkription
AU2014274840B2 (en) * 2013-06-05 2020-03-12 Duke University RNA-guided gene editing and gene regulation
CA2942268A1 (fr) * 2014-03-12 2015-09-17 Precision Biosciences, Inc. Deletion d'exon du gene codant la dystrophine au moyen de nucleases genetiquement modifiees
EP3180034B1 (fr) * 2014-08-11 2022-04-20 The Board of Regents of The University of Texas System Prévention de la dystrophie musculaire par édition de gène médiée par crispr/cas9
US20170266320A1 (en) * 2014-12-01 2017-09-21 President And Fellows Of Harvard College RNA-Guided Systems for In Vivo Gene Editing
IL286316B2 (en) * 2015-01-16 2023-03-01 Univ Washington New micro-dystrophins and related method of use
CA2981715A1 (fr) * 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Arn guides chimiquement modifies pour la regulation genetique mediee par crispr/cas
WO2016174056A1 (fr) * 2015-04-27 2016-11-03 Genethon Compositions et méthodes pour le traitement de troubles dus à l'expansion de répétition des nucléotides
CN108513546A (zh) * 2015-10-28 2018-09-07 克里斯珀医疗股份公司 用于治疗杜氏肌营养不良症的材料和方法
BR112018011133A2 (pt) * 2015-11-30 2018-11-21 Univ Duke alvos terapêuticos para a correção do gene humano de distrofina por edição de gene e métodos de uso
US20170362635A1 (en) * 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes

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ECSP19056408A (es) 2019-09-30
DOP2019000179A (es) 2019-11-15
CN110506115A (zh) 2019-11-26
JP2020503869A (ja) 2020-02-06
EP3565897A1 (fr) 2019-11-13
SG11201906147VA (en) 2019-08-27
JOP20190166A1 (ar) 2019-07-02
PH12019501561A1 (en) 2019-09-09
US20190338311A1 (en) 2019-11-07
BR112019013962A2 (pt) 2020-02-11
WO2018129296A1 (fr) 2018-07-12
KR102606174B1 (ko) 2023-11-27
MX2019008064A (es) 2020-07-20
PE20191357A1 (es) 2019-10-01
KR20190100967A (ko) 2019-08-29
CR20190326A (es) 2019-10-02
AU2018205521A1 (en) 2019-07-18
CO2019008181A2 (es) 2019-10-31
CA3048635A1 (fr) 2018-07-12
IL267786A (en) 2019-09-26
CL2019001882A1 (es) 2019-10-04

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