CO2019008181A2 - 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
CO2019008181A2
CO2019008181A2 CO2019008181A CO2019008181A CO2019008181A2 CO 2019008181 A2 CO2019008181 A2 CO 2019008181A2 CO 2019008181 A CO2019008181 A CO 2019008181A CO 2019008181 A CO2019008181 A CO 2019008181A CO 2019008181 A2 CO2019008181 A2 CO 2019008181A2
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CO
Colombia
Prior art keywords
cas9
crispr
modifications
leader sequences
exon skipping
Prior art date
Application number
CO2019008181A
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English (en)
Inventor
Leonela Amoasii
Eric Olson
Original Assignee
Univ Texas
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Publication date
Application filed by Univ Texas filed Critical Univ Texas
Publication of CO2019008181A2 publication Critical patent/CO2019008181A2/es

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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 ΔEx50-MD.
CO2019008181A 2017-01-05 2019-07-29 Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples CO2019008181A2 (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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CO2019008181A2 true CO2019008181A2 (es) 2019-10-31

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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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WO2019246480A1 (en) * 2018-06-21 2019-12-26 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 The Regents of the University of 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
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EP4053285A4 (en) * 2019-10-29 2024-06-19 Genkore Inc GUIDE RNA MODIFIED TO INCREASE THE EFFICIENCY OF CRISPR/CAS12F1 SYSTEM, AND ITS USE
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
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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
WO2023172116A1 (ko) * 2022-03-10 2023-09-14 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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