ECSP19056408A - 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
ECSP19056408A
ECSP19056408A ECSENADI201956408A ECDI201956408A ECSP19056408A EC SP19056408 A ECSP19056408 A EC SP19056408A EC SENADI201956408 A ECSENADI201956408 A EC SENADI201956408A EC DI201956408 A ECDI201956408 A EC DI201956408A EC SP19056408 A ECSP19056408 A EC SP19056408A
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Ecuador
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cas9
crispr
modifications
guide sequences
dmd
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ECSENADI201956408A
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English (en)
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Abstract

La edición genómica mediada por CRISPR/Cas9 tiene un potencial clínico para el tratamiento de enfermedades genéticas, como la distrofia muscular de Duchenne (DMD), que es causada por mutaciones en el gen de la distrofina. En la presente, mediante el uso de tres promotores para dirigir la expresión del mismo ARN guía de DMD, se logró una forma más sólida y segura de edición genómica en un modelo de ratón humanizado para DMD con una eliminación en el exón 50 y en un modelo de perro 916;Ex50-MD.
ECSENADI201956408A 2017-01-05 2019-08-06 Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples ECSP19056408A (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)

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ECSP19056408A true ECSP19056408A (es) 2019-09-30

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ECSENADI201956408A ECSP19056408A (es) 2017-01-05 2019-08-06 Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples

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

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WO2019136216A1 (en) * 2018-01-05 2019-07-11 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
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WO2020210776A1 (en) * 2019-04-12 2020-10-15 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
EP3952925A4 (en) * 2019-04-12 2024-01-24 Univ California COMPOSITIONS AND METHODS FOR MODIFYING DYSTROPHIN GENES
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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
US20240091379A1 (en) * 2019-10-11 2024-03-21 Yale University Compositions and methods for upregulating isoforms of dystrophin as therapy for duchenne muscular dystrophy (dmd)
WO2021086083A2 (ko) * 2019-10-29 2021-05-06 주식회사 진코어 CRISPR/Cas12f1 시스템 효율화를 위한 엔지니어링 된 가이드 RNA 및 그 용도
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
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
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

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US20170362635A1 (en) 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes

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