AU2016362282B2 - Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use - Google Patents

Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use Download PDF

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AU2016362282B2
AU2016362282B2 AU2016362282A AU2016362282A AU2016362282B2 AU 2016362282 B2 AU2016362282 B2 AU 2016362282B2 AU 2016362282 A AU2016362282 A AU 2016362282A AU 2016362282 A AU2016362282 A AU 2016362282A AU 2016362282 B2 AU2016362282 B2 AU 2016362282B2
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gene
correction
methods
therapeutic targets
human dystrophin
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AU2016362282A1 (en
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Charles A. Gersbach
Jacqueline N. Robinson-Hamm
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Duke University
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Duke University
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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AU2016362282A 2015-11-30 2016-11-30 Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use Active AU2016362282B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562260712P 2015-11-30 2015-11-30
US62/260,712 2015-11-30
US201662330336P 2016-05-02 2016-05-02
US62/330,336 2016-05-02
PCT/US2016/064285 WO2017095967A2 (en) 2015-11-30 2016-11-30 Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use

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AU2016362282A1 AU2016362282A1 (en) 2018-05-10
AU2016362282B2 true AU2016362282B2 (en) 2023-03-16

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US (1) US12214054B2 (enExample)
EP (2) EP4644567A2 (enExample)
JP (3) JP7108307B2 (enExample)
KR (2) KR20250044471A (enExample)
CN (2) CN108779466B (enExample)
AU (1) AU2016362282B2 (enExample)
CA (1) CA3001623A1 (enExample)
EA (1) EA201891317A3 (enExample)
IL (1) IL259100B2 (enExample)
MX (3) MX2018005377A (enExample)
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EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US11369692B2 (en) 2015-10-28 2022-06-28 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of Duchenne Muscular Dystrophy
CN108779466B (zh) * 2015-11-30 2024-03-29 杜克大学 用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
ES2957660T3 (es) * 2016-05-05 2024-01-23 Univ Duke Composiciones relacionadas con crispr/cas para tratar la distrofia muscular de duchenne
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
JOP20190166A1 (ar) * 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
US10687520B2 (en) 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44
EP3668983A1 (en) * 2017-08-18 2020-06-24 The Board of Regents of The University of Texas System Exon deletion correction of duchenne muscular dystrophy mutations in the dystrophin actin binding domain 1 using crispr genome editing
EP3707155A2 (en) * 2017-11-09 2020-09-16 Vertex Pharmaceuticals Incorporated Crispr/cas systems for treatment of dmd
US11891635B2 (en) 2017-12-21 2024-02-06 Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft Nucleic acid sequence replacement by NHEJ
BR112020019079A2 (pt) * 2018-03-23 2020-12-29 Massachusetts Eye And Ear Infirmary Abordagem de salto de éxon mediada por crispr/cas9 para síndrome de usher associada a ush2a
WO2019209777A1 (en) 2018-04-23 2019-10-31 The Curators Of The University Of Missouri Improved crispr therapy
JP7788855B2 (ja) * 2018-12-12 2025-12-19 ソリッド・バイオサイエンシーズ・インコーポレーテッド 筋ジストロフィーの治療のための併用療法
WO2020210776A1 (en) * 2019-04-12 2020-10-15 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
BR112021020515A2 (pt) * 2019-04-14 2022-01-04 Univ Duke Deleção mediada por vetor de aav de hotspot mutacional grande para o tratamento de distrofia muscular de duchenne
EP3966327A1 (en) * 2019-05-08 2022-03-16 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
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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