PE20191357A1 - Estrategia optimizada para modificaciones de omision de exones mediante el uso de crispr/cas9 con secuencias guia triples - Google Patents

Estrategia optimizada para modificaciones de omision de exones mediante el uso de crispr/cas9 con secuencias guia triples

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Publication number
PE20191357A1
PE20191357A1 PE2019001384A PE2019001384A PE20191357A1 PE 20191357 A1 PE20191357 A1 PE 20191357A1 PE 2019001384 A PE2019001384 A PE 2019001384A PE 2019001384 A PE2019001384 A PE 2019001384A PE 20191357 A1 PE20191357 A1 PE 20191357A1
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PE
Peru
Prior art keywords
cas9
crispr
modifications
guide sequences
dmd
Prior art date
Application number
PE2019001384A
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English (en)
Inventor
Leonela Amoasii
Eric Olson
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Univ Texas
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Application filed by Univ Texas filed Critical Univ Texas
Publication of PE20191357A1 publication Critical patent/PE20191357A1/es

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Abstract

La edicion genomica mediada por CRISPR/Cas9 tiene un potencial clinico para el tratamiento de enfermedades geneticas, 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 expresion del mismo ARN guia de DMD, se logro una forma mas solida y segura de edicion genomica en un modelo de raton humanizado para DMD con una eliminacion en el exon 50 y en un modelo de perro DELTAEx50-MD
PE2019001384A 2017-01-05 2018-01-05 Estrategia optimizada para modificaciones de omision de exones mediante el uso de crispr/cas9 con secuencias guia triples PE20191357A1 (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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PE20191357A1 true PE20191357A1 (es) 2019-10-01

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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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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
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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JP2022526669A (ja) * 2019-04-12 2022-05-25 デューク ユニバーシティ ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物
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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
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)
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
US11638761B2 (en) 2021-07-09 2023-05-02 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
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

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US20190338311A1 (en) 2019-11-07
BR112019013962A2 (pt) 2020-02-11
WO2018129296A1 (en) 2018-07-12
KR102606174B1 (ko) 2023-11-27
MX2019008064A (es) 2020-07-20
MA47239A (fr) 2019-11-13
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AU2018205521A1 (en) 2019-07-18
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