CL2019001882A1 - Estrategia optimizada para modificaciones de omisión de exón utilizando crispr/cas9 con secuencias de guía triple. - Google Patents

Estrategia optimizada para modificaciones de omisión de exón utilizando crispr/cas9 con secuencias de guía triple.

Info

Publication number
CL2019001882A1
CL2019001882A1 CL2019001882A CL2019001882A CL2019001882A1 CL 2019001882 A1 CL2019001882 A1 CL 2019001882A1 CL 2019001882 A CL2019001882 A CL 2019001882A CL 2019001882 A CL2019001882 A CL 2019001882A CL 2019001882 A1 CL2019001882 A1 CL 2019001882A1
Authority
CL
Chile
Prior art keywords
cas9
crispr
modifications
exon skipping
guide sequences
Prior art date
Application number
CL2019001882A
Other languages
English (en)
Spanish (es)
Inventor
Leonela Amoasil
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 CL2019001882A1 publication Critical patent/CL2019001882A1/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
    • C07K14/4708Duchenne dystrophy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0652Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657Cardiomyocytes; Heart cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0652Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0658Skeletal muscle cells, e.g. myocytes, myotubes, myoblasts
    • C12N5/0659Satellite cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0652Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/066Tenocytes; Tendons, Ligaments
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0652Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0661Smooth muscle cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0696Artificially induced pluripotent stem cells, e.g. iPS
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/90Vectors containing a transposable element

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • 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)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Rheumatology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Developmental Biology & Embryology (AREA)
  • Mycology (AREA)
  • Virology (AREA)
  • Transplantation (AREA)
  • Crystallography & Structural Chemistry (AREA)
CL2019001882A 2017-01-05 2019-07-05 Estrategia optimizada para modificaciones de omisión de exón utilizando crispr/cas9 con secuencias de guía triple. CL2019001882A1 (es)

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
CL2019001882A1 true CL2019001882A1 (es) 2019-10-04

Family

ID=61193018

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2019001882A CL2019001882A1 (es) 2017-01-05 2019-07-05 Estrategia optimizada para modificaciones de omisión de exón utilizando crispr/cas9 con secuencias de guía triple.

Country Status (22)

Country Link
US (1) US20190338311A1 (OSRAM)
EP (1) EP3565897B1 (OSRAM)
JP (1) JP2020503869A (OSRAM)
KR (1) KR102606174B1 (OSRAM)
CN (1) CN110506115A (OSRAM)
AU (1) AU2018205521A1 (OSRAM)
BR (1) BR112019013962A2 (OSRAM)
CA (1) CA3048635A1 (OSRAM)
CL (1) CL2019001882A1 (OSRAM)
CO (1) CO2019008181A2 (OSRAM)
CR (1) CR20190326A (OSRAM)
DK (1) DK3565897T3 (OSRAM)
DO (1) DOP2019000179A (OSRAM)
EC (1) ECSP19056408A (OSRAM)
IL (1) IL267786A (OSRAM)
JO (1) JOP20190166A1 (OSRAM)
MA (1) MA47239A (OSRAM)
MX (1) MX2019008064A (OSRAM)
PE (1) PE20191357A1 (OSRAM)
PH (1) PH12019501561A1 (OSRAM)
SG (1) SG11201906147VA (OSRAM)
WO (1) WO2018129296A1 (OSRAM)

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
AU2016362282B2 (en) 2015-11-30 2023-03-16 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
KR102758434B1 (ko) * 2017-03-30 2025-01-21 고쿠리츠 다이가쿠 호진 교토 다이가쿠 게놈 편집에 의한 엑손 스키핑 유도 방법
EP3735462A1 (en) * 2018-01-05 2020-11-11 The Board of Regents of The University of Texas System Therapeutic crispr/cas9 compositions and methods of use
US20210261962A1 (en) * 2018-06-21 2021-08-26 The Board Of Regents Of The University Of Texas System Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2020122104A1 (ja) * 2018-12-11 2020-06-18 国立大学法人京都大学 ゲノムdnaに欠失を誘導する方法
WO2020142714A1 (en) * 2019-01-04 2020-07-09 Exonics Therapeutics, Inc. Aav expression cassette and aav vectors comprising the same
EP3935179A4 (en) * 2019-03-07 2022-11-23 The Trustees of Columbia University in the City of New York Rna-guided dna integration using tn7-like transposons
EP3952884A4 (en) * 2019-04-12 2023-03-22 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
EP3952925A4 (en) * 2019-04-12 2024-01-24 The Regents of the University of California COMPOSITIONS AND METHODS FOR MODIFYING DYSTROPHIN GENES
EP3966327A1 (en) 2019-05-08 2022-03-16 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of 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 (en) * 2019-10-11 2021-04-15 Yale University Compositions and methods for upregulating isoforms of dystrophin as therapy for duchenne muscular dystrophy (dmd)
KR102455623B1 (ko) * 2019-10-29 2022-10-18 주식회사 진코어 CRISPR/Cas12f1 시스템 효율화를 위한 엔지니어링 된 가이드 RNA 및 그 용도
CN116096394A (zh) * 2020-02-13 2023-05-09 特纳亚治疗股份有限公司 用于治疗心脏病的基因疗法载体
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
JP2023515709A (ja) * 2020-04-27 2023-04-13 デューク ユニバーシティ 筋肉特異的プロモーターをコードするaavベクターを使用するインビボでの衛星細胞の遺伝子編集
CN112063621B (zh) * 2020-09-02 2022-06-28 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
JP2023540783A (ja) * 2020-09-09 2023-09-26 バーテックス ファーマシューティカルズ インコーポレイテッド デュシェンヌ型筋ジストロフィーの治療のための組成物及び方法
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023159103A1 (en) * 2022-02-17 2023-08-24 The Board Of Regents Of The University Of Texas System CRISPR/SpCas9 VARIANT AND METHODS FOR ENHANCED CORRECTON OF DUCHENNE MUSCULAR DYSTROPHY MUTATIONS
KR20230134097A (ko) * 2022-03-10 2023-09-20 주식회사 진코어 Nhej 복구 경로 조절을 통해 핵산 세그먼트의 결실 효율을 증가시키기 위한 조성물 및 방법
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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1997745A (zh) * 2004-04-02 2007-07-11 得克萨斯州大学系统董事会 癌症特异性启动子
SI3401400T1 (sl) 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
KR20230136697A (ko) * 2013-06-05 2023-09-26 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
DK3116533T3 (da) * 2014-03-12 2020-08-24 Prec Biosciences Inc Deletion af dystrophingenexon under anvendelse af genmanipulerede nukleaser
CA2959130A1 (en) * 2014-08-11 2016-02-18 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cas9-mediated gene editing
US20170266320A1 (en) * 2014-12-01 2017-09-21 President And Fellows Of Harvard College RNA-Guided Systems for In Vivo Gene Editing
CA2972811A1 (en) * 2015-01-16 2016-07-21 University Of Washington Novel micro-dystrophins and related methods of use
WO2016164356A1 (en) * 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
WO2016174056A1 (en) * 2015-04-27 2016-11-03 Genethon Compositions and methods for the treatment of nucleotide repeat expansion disorders
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
AU2016362282B2 (en) * 2015-11-30 2023-03-16 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
US20170362635A1 (en) * 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes

Also Published As

Publication number Publication date
JOP20190166A1 (ar) 2019-07-02
ECSP19056408A (es) 2019-09-30
IL267786A (en) 2019-09-26
MA47239A (fr) 2019-11-13
DK3565897T3 (da) 2025-02-10
MX2019008064A (es) 2020-07-20
DOP2019000179A (es) 2019-11-15
CO2019008181A2 (es) 2019-10-31
CR20190326A (es) 2019-10-02
KR20190100967A (ko) 2019-08-29
WO2018129296A1 (en) 2018-07-12
EP3565897B1 (en) 2024-12-11
AU2018205521A1 (en) 2019-07-18
CA3048635A1 (en) 2018-07-12
CN110506115A (zh) 2019-11-26
JP2020503869A (ja) 2020-02-06
BR112019013962A2 (pt) 2020-02-11
PH12019501561A1 (en) 2019-09-09
PE20191357A1 (es) 2019-10-01
EP3565897A1 (en) 2019-11-13
US20190338311A1 (en) 2019-11-07
SG11201906147VA (en) 2019-08-27
KR102606174B1 (ko) 2023-11-27

Similar Documents

Publication Publication Date Title
CL2019001882A1 (es) Estrategia optimizada para modificaciones de omisión de exón utilizando crispr/cas9 con secuencias de guía triple.
ZA202206102B (en) Excision of retroviral nucleic acid sequences
SG10201809290SA (en) Compositions and Methods to Treating Hemoglobinopathies
MX2020012560A (es) Metodos para tratar infecciones por el virus de filoviridae.
HK1258823A1 (zh) 用於治疗肌营养不良的方法
MX2017000143A (es) Composiciones enriquecidas estereoquimicamente para administracion de acidos nucleicos.
SG10201804715WA (en) Crispr hybrid dna/rna polynucleotides and methods of use
CO2017007335A2 (es) Supresión del gen de la huntingtina inducida por la arni
BR112017024514A2 (pt) edição genética de mutações intrônicas profundas
CL2016001963A1 (es) Heteroarilos inhibidores de syk
MY197236A (en) 2'-substituted-n6-substituted purine nucleotides for rna virus treatment
MX377983B (es) Análogos de nucleótidos.
BR112015023489A2 (pt) uso de rnas-guia truncads (tru - grnas) para aumentar a especificidade de edição de genoma com rna guiado
BR112015022522A2 (pt) engenharia de genomas de planta usando sistemas crispr/cas
MX2019014100A (es) Variantes de endonucleasas cblb, composiciones y metodos de uso.
JP2016528887A5 (OSRAM)
CO2017001884A2 (es) Polimorfos de selinexor
JP2016526529A5 (OSRAM)
AR098996A1 (es) Formulación compuesta para la administración por vía oral que comprende ezetimiba y rosuvastatina
PH12016502555A1 (en) Methods of preparing substituted nucleotide analogs
MX2020012652A (es) Oligonucleotido bicatenario especifico del gen de anfirregulina y composicion para prevenir y tratar enfermedades relacionadas con la fibrosis y enfermedades respiratorias, que comprende el mismo.
JO3571B1 (ar) كحولات ثلاثي فلوروميثيل كمُعدلات للمستقبل النووي جاما تي المرتبط بحمض الريتيونَويك ror-gamma-t
CL2016002879A1 (es) Composiciones farmacéuticas para tratar enfermedades infecciosas
MX2016009747A (es) Vacunas contra el reovirus aviar.
BR112016017592A2 (pt) Microrganismos que apresentam produtividade aumentada de l-aminoácidos e processo para produção de l-aminoácidos utilizando estes