AU2018205521A1 - Optimized strategy for exon skipping modifications using CRISPR/Cas9 with triple guide sequences - Google Patents

Optimized strategy for exon skipping modifications using CRISPR/Cas9 with triple guide sequences Download PDF

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Publication number
AU2018205521A1
AU2018205521A1 AU2018205521A AU2018205521A AU2018205521A1 AU 2018205521 A1 AU2018205521 A1 AU 2018205521A1 AU 2018205521 A AU2018205521 A AU 2018205521A AU 2018205521 A AU2018205521 A AU 2018205521A AU 2018205521 A1 AU2018205521 A1 AU 2018205521A1
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sequence encoding
promoter
sequence
nucleic acid
vector
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Leonela AMOASII
Eric Olson
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University of Texas System
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University of Texas System
University of Texas at Austin
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AU2018205521A 2017-01-05 2018-01-05 Optimized strategy for exon skipping modifications using CRISPR/Cas9 with triple guide sequences Abandoned AU2018205521A1 (en)

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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AU2018205521A1 true AU2018205521A1 (en) 2019-07-18

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AU2018205521A Abandoned AU2018205521A1 (en) 2017-01-05 2018-01-05 Optimized strategy for exon skipping modifications using CRISPR/Cas9 with triple guide sequences

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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)
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JP2023515709A (ja) * 2020-04-27 2023-04-13 デューク ユニバーシティ 筋肉特異的プロモーターをコードするａａｖベクターを使用するインビボでの衛星細胞の遺伝子編集
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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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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
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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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