MX2019008064A - 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
MX2019008064A
MX2019008064A MX2019008064A MX2019008064A MX2019008064A MX 2019008064 A MX2019008064 A MX 2019008064A MX 2019008064 A MX2019008064 A MX 2019008064A MX 2019008064 A MX2019008064 A MX 2019008064A MX 2019008064 A MX2019008064 A MX 2019008064A
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MX
Mexico
Prior art keywords
cas9
crispr
modifications
exon skipping
guide sequences
Prior art date
Application number
MX2019008064A
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English (en)
Inventor
Eric Olson
Leonela Amoasii
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Univ Texas
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Publication of MX2019008064A publication Critical patent/MX2019008064A/es

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Abstract

La edición genómica mediada por CRISPR/Casg tiene un potencial tánico 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 gula 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 ?Ex50-MD.
MX2019008064A 2017-01-05 2018-01-05 Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr / cas9 con secuencias guía triples. MX2019008064A (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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MX2019008064A true MX2019008064A (es) 2020-07-20

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MX2019008064A MX2019008064A (es) 2017-01-05 2018-01-05 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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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)
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
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
KR20230134098A (ko) * 2022-03-10 2023-09-20 주식회사 진코어 듀센 근이영양증 치료를 위한 유전자 편집 시스템 및 이를 이용한 질병 치료 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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