DOP2019000179A - Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples - Google Patents

Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples

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Publication number
DOP2019000179A
DOP2019000179A DO2019000179A DO2019000179A DOP2019000179A DO P2019000179 A DOP2019000179 A DO P2019000179A DO 2019000179 A DO2019000179 A DO 2019000179A DO 2019000179 A DO2019000179 A DO 2019000179A DO P2019000179 A DOP2019000179 A DO P2019000179A
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Dominican Republic
Prior art keywords
cas9
crispr
modifications
guide sequences
dmd
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DO2019000179A
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English (en)
Spanish (es)
Inventor
Amoasii Leonela
Olson Eric
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Publication of DOP2019000179A publication Critical patent/DOP2019000179A/es

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DO2019000179A 2017-01-05 2019-07-05 Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples DOP2019000179A (es)

Applications Claiming Priority (4)

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
PCT/US2018/012558 WO2018129296A1 (en) 2017-01-05 2018-01-05 Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences

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DOP2019000179A true DOP2019000179A (es) 2019-11-15

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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
WO2013163628A2 (en) 2012-04-27 2013-10-31 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
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
WO2017095967A2 (en) 2015-11-30 2017-06-08 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
WO2018179578A1 (ja) * 2017-03-30 2018-10-04 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 (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
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
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP7395159B2 (ja) * 2018-12-11 2023-12-11 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
WO2020210776A1 (en) * 2019-04-12 2020-10-15 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
US20220177876A1 (en) * 2019-04-12 2022-06-09 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
MX2022009883A (es) * 2020-02-13 2022-10-03 Tenaya Therapeutics Inc Vectores de genoterapia para tratar enfermedades cardíacas.
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
US20230348870A1 (en) * 2020-04-27 2023-11-02 Duke University Gene editing of satellite cells in vivo using aav vectors encoding muscle-specific promoters
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
EP4211242A1 (en) * 2020-09-09 2023-07-19 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of duchenne muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
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
MX2024000489A (es) 2021-07-09 2024-04-09 Dyne Therapeutics Inc Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías.
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
WO2023172115A1 (ko) * 2022-03-10 2023-09-14 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
ATE557094T1 (de) * 2004-04-02 2012-05-15 Univ Texas Krebsspezifische promotoren
MY189533A (en) 2012-05-25 2022-02-16 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
KR102733409B1 (ko) * 2013-06-05 2024-11-21 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
WO2015138739A2 (en) * 2014-03-12 2015-09-17 Precision Biosciences, Inc. Dystrophin gene oxon deletion using engineered nucleases
JP6837429B2 (ja) * 2014-08-11 2021-03-03 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム Crispr/cas9媒介遺伝子編集による筋ジストロフィーの予防
US20170266320A1 (en) * 2014-12-01 2017-09-21 President And Fellows Of Harvard College RNA-Guided Systems for In Vivo Gene Editing
EP4599851A3 (en) * 2015-01-16 2025-12-31 University of Washington NEW MICRO-DYSTROPHINS AND ASSOCIATED METHODS OF USE
EP3280803B1 (en) * 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
US11104897B2 (en) * 2015-04-27 2021-08-31 Genethon Compositions and methods for the treatment of nucleotide repeat expansion disorders
EP3368063B1 (en) * 2015-10-28 2023-09-06 Vertex Pharmaceuticals Inc. Materials and methods for treatment of duchenne muscular dystrophy
WO2017095967A2 (en) * 2015-11-30 2017-06-08 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

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