CR20190326A - 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
CR20190326A
CR20190326A CR20190326A CR20190326A CR20190326A CR 20190326 A CR20190326 A CR 20190326A CR 20190326 A CR20190326 A CR 20190326A CR 20190326 A CR20190326 A CR 20190326A CR 20190326 A CR20190326 A CR 20190326A
Authority
CR
Costa Rica
Prior art keywords
dmd
cas9
crispr
modifications
exon skipping
Prior art date
Application number
CR20190326A
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English (en)
Spanish (es)
Inventor
Eric Olson
Leonela Amoasii
Original Assignee
Univ Texas
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by Univ Texas filed Critical Univ Texas
Publication of CR20190326A publication Critical patent/CR20190326A/es

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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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CR20190326A 2017-01-05 2018-01-05 Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples CR20190326A (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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CR20190326A true CR20190326A (es) 2019-10-02

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US (1) US20190338311A1 (enExample)
EP (1) EP3565897B1 (enExample)
JP (1) JP2020503869A (enExample)
KR (1) KR102606174B1 (enExample)
CN (1) CN110506115A (enExample)
AU (1) AU2018205521A1 (enExample)
BR (1) BR112019013962A2 (enExample)
CA (1) CA3048635A1 (enExample)
CL (1) CL2019001882A1 (enExample)
CO (1) CO2019008181A2 (enExample)
CR (1) CR20190326A (enExample)
DK (1) DK3565897T3 (enExample)
DO (1) DOP2019000179A (enExample)
EC (1) ECSP19056408A (enExample)
IL (1) IL267786A (enExample)
JO (1) JOP20190166A1 (enExample)
MA (1) MA47239A (enExample)
MX (1) MX2019008064A (enExample)
PE (1) PE20191357A1 (enExample)
PH (1) PH12019501561A1 (enExample)
SG (1) SG11201906147VA (enExample)
WO (1) WO2018129296A1 (enExample)

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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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EP4125349A4 (en) * 2020-04-27 2024-07-10 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
JP2023540783A (ja) * 2020-09-09 2023-09-26 バーテックス ファーマシューティカルズ インコーポレイテッド デュシェンヌ型筋ジストロフィーの治療のための組成物及び方法
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EP4479536A1 (en) * 2022-02-17 2024-12-25 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
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BR112019013962A2 (pt) 2020-02-11
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