CN110506115A - 使用CRISPR/Cas9和三重指导序列进行外显子跳读修饰的优化策略 - Google Patents

使用CRISPR/Cas9和三重指导序列进行外显子跳读修饰的优化策略 Download PDF

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CN110506115A
CN110506115A CN201880010288.7A CN201880010288A CN110506115A CN 110506115 A CN110506115 A CN 110506115A CN 201880010288 A CN201880010288 A CN 201880010288A CN 110506115 A CN110506115 A CN 110506115A
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sequence encoding
promoter
sequence
nucleic acid
vector
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利奥内拉·阿莫阿西
埃里克·奥尔松
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University of Texas System
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University of Texas System
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CN201880010288.7A 2017-01-05 2018-01-05 使用CRISPR/Cas9和三重指导序列进行外显子跳读修饰的优化策略 Pending CN110506115A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762442606P 2017-01-05 2017-01-05
US62/442,606 2017-01-05
US201762544449P 2017-08-11 2017-08-11
US62/544,449 2017-08-11
US201762596298P 2017-12-08 2017-12-08
US62/596,298 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

Publications (1)

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CN110506115A true CN110506115A (zh) 2019-11-26

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CN201880010288.7A Pending CN110506115A (zh) 2017-01-05 2018-01-05 使用CRISPR/Cas9和三重指导序列进行外显子跳读修饰的优化策略

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

Cited By (2)

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CN111172191A (zh) * 2020-02-21 2020-05-19 浙江大学 一种高效基因敲除载体及其应用
CN112063621A (zh) * 2020-09-02 2020-12-11 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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EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
EP3617311B1 (en) * 2017-03-30 2024-11-20 Kyoto University Method for inducing exon skipping by genome editing
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
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
EP3810775A1 (en) * 2018-06-21 2021-04-28 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
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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WO2020122104A1 (ja) 2018-12-11 2020-06-18 国立大学法人京都大学 ゲノムｄｎａに欠失を誘導する方法
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BR112021017655A2 (pt) * 2019-03-07 2021-11-16 Univ Columbia Método e sistema de integração de dna guiada por rna usando transposons tipo tn7
US20220177876A1 (en) * 2019-04-12 2022-06-09 The Regents Of The University Of California Compositions and Methods for Modifying Dystrophin Genes
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
US20210047649A1 (en) 2019-05-08 2021-02-18 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
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
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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
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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
AU2021342057A1 (en) * 2020-09-09 2023-04-06 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of duchenne muscular dystrophy
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JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11969475B2 (en) 2021-07-09 2024-04-30 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
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