CR20190326A - Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples - Google Patents

Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples

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Publication number
CR20190326A
CR20190326A CR20190326A CR20190326A CR20190326A CR 20190326 A CR20190326 A CR 20190326A CR 20190326 A CR20190326 A CR 20190326A CR 20190326 A CR20190326 A CR 20190326A CR 20190326 A CR20190326 A CR 20190326A
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Costa Rica
Prior art keywords
dmd
cas9
crispr
modifications
exon skipping
Prior art date
Application number
CR20190326A
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English (en)
Inventor
Eric Olson
Leonela Amoasii
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Univ Texas
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Publication date
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Publication of CR20190326A publication Critical patent/CR20190326A/es

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Abstract

La edición genómica mediada por CRISPR/Cas9 tiene un potencial clínico para el tratamiento de enfermedades genéticas, como la distrofia muscular de Duchenne (DMD), que es causada por mutaciones en el gen de la distrofina. En la presente, mediante el uso de tres promotores para dirigir la expresión del mismo ARN guía de DMD, se logró una forma más sólida y segura de edición 5 genómica en un modelo de ratón humanizado para DMD con una eliminación en el exón 50 y en un modelo de perro ¿Ex50-MD.CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. Here, using three promoters to drive expression of the same DMD guide RNA, a more robust and safe form of genome editing was achieved in a humanized mouse model for DMD with a deletion in exon 50, and in a ¿Ex50-MD Dog.
CR20190326A 2017-01-05 2018-01-05 Estrategia optimizada para modificaciones de omisión de exones mediante el uso de crispr/cas9 con secuencias guía triples CR20190326A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762442606P 2017-01-05 2017-01-05
US201762544449P 2017-08-11 2017-08-11
US201762596298P 2017-12-08 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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CR20190326A true CR20190326A (es) 2019-10-02

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

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013163628A2 (en) 2012-04-27 2013-10-31 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
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
WO2017095967A2 (en) 2015-11-30 2017-06-08 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
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
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
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
JP7611825B2 (ja) 2018-08-02 2025-01-10 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それらの使用
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral 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
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
EP3935179A4 (en) 2019-03-07 2022-11-23 The Trustees of Columbia University in the City of New York RNA-GUIDED DNA INTEGRATION USING TN7-LIKE TRANSPOSONS
US20220177879A1 (en) * 2019-04-12 2022-06-09 Duke University Crispr/cas-based base editing composition for restoring dystrophin function
WO2020210214A1 (en) * 2019-04-12 2020-10-15 The Regents Of The University Of California Compositions and methods for modifying dystrophin genes
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)
JP7799912B2 (ja) * 2019-10-29 2026-01-16 ゲンコレ インコーポレイテッド 最適化されたＣＲＩＳＰＲ／Ｃａｓ１２ｆ１システムのための人工ガイドＲＮＡおよびその使用
MX2022009883A (es) * 2020-02-13 2022-10-03 Tenaya Therapeutics Inc Vectores de genoterapia para tratar enfermedades cardíacas.
CN111172191B (zh) * 2020-02-21 2020-12-22 浙江大学 一种高效基因敲除载体及其应用
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
CN112063621B (zh) * 2020-09-02 2022-06-28 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
MX2023002755A (es) * 2020-09-09 2023-05-19 Vertex Pharma Composiciones y metodos para el tratamiento de la distrofia muscular de duchenne.
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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
CN117980006A (zh) 2021-07-09 2024-05-03 达因疗法公司 用于治疗肌养蛋白病的肌肉靶向复合物和制剂
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
WO2023172115A1 (ko) * 2022-03-10 2023-09-14 주식회사 진코어 Nhej 복구 경로 조절을 통해 핵산 세그먼트의 결실 효율을 증가시키기 위한 조성물 및 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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2385492T3 (es) * 2004-04-02 2012-07-25 Board Of Regents, The University Of Texas System Promotores específicos de cáncer
LT3401400T (lt) 2012-05-25 2019-06-10 The Regents Of The University Of California Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui
ES3029138T3 (en) * 2013-06-05 2025-06-23 Univ Duke Rna-guided gene editing and gene regulation
AU2015229299A1 (en) * 2014-03-12 2016-09-22 Precision Biosciences, Inc. Dystrophin gene exon deletion using engineered nucleases
CN106714845A (zh) * 2014-08-11 2017-05-24 得克萨斯州大学系统董事会 通过crispr/cas9介导的基因编辑预防肌营养不良
WO2016089866A1 (en) * 2014-12-01 2016-06-09 President And Fellows Of Harvard College Rna-guided systems for in vivo gene editing
JP6832280B2 (ja) * 2015-01-16 2021-02-24 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ 新規のマイクロジストロフィンおよび使用の関連する方法
KR102888521B1 (ko) * 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
EP3289081B1 (en) * 2015-04-27 2019-03-27 Genethon Compositions and methods for the treatment of nucleotide repeat expansion disorders
AU2016344609B2 (en) * 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
WO2017095967A2 (en) * 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
US20170362635A1 (en) * 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes

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