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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The 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)
CL (1) CL2019001882A1 (enExample)
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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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WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD 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
CN110892069B (zh) * 2017-03-30 2024-02-09 国立大学法人京都大学 基于基因组编辑的外显子跳跃诱导方法
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
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
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
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
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EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
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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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
WO2020210214A1 (en) * 2019-04-12 2020-10-15 The Regents Of The University Of California Compositions and methods for modifying dystrophin genes
JP7881154B2 (ja) * 2019-04-12 2026-06-29 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
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
EP3966327A1 (en) 2019-05-08 2022-03-16 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
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RU2722934C1 (ru) 2019-06-11 2020-06-05 Автономная некоммерческая образовательная организация высшего образования Сколковский институт науки и технологий Средство разрезания днк на основе cas9 белка из бактерии pasteurella pneumotropica
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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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US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
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