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
promoter
coding
nucleic acid
carrier
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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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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

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CN110506115A true CN110506115A (zh) 2019-11-26

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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)
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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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EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
MX2018005377A (es) 2015-11-30 2018-11-09 Univ Duke Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
KR102758434B1 (ko) * 2017-03-30 2025-01-21 고쿠리츠 다이가쿠 호진 교토 다이가쿠 게놈 편집에 의한 엑손 스키핑 유도 방법
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
JP2021533200A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
EP3896158A4 (en) * 2018-12-11 2022-11-02 Kyoto University METHODS OF INDUCING A DELETION IN GENOMIC DNA
WO2020142714A1 (en) * 2019-01-04 2020-07-09 Exonics Therapeutics, Inc. Aav expression cassette and aav vectors comprising the same
WO2020181264A1 (en) * 2019-03-07 2020-09-10 The Trustees Of Columbia University In The City Of New York Rna-guided dna integration using tn7-like transposons
EP3952925A4 (en) * 2019-04-12 2024-01-24 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
WO2020225606A1 (en) 2019-05-08 2020-11-12 Crispr Therapeutics Ag 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
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)
EP4053285A4 (en) * 2019-10-29 2024-06-19 Genkore Inc GUIDE RNA MODIFIED TO INCREASE THE EFFICIENCY OF CRISPR/CAS12F1 SYSTEM, AND ITS USE
MY209860A (en) * 2020-02-13 2025-08-07 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease
EP4125349A4 (en) * 2020-04-27 2024-07-10 Duke University GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
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
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
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
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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