Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression-Reference-Cited by-同舟云学术

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Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression

Published:2021-03-16 Issue:5 Volume:13 Page:
ISSN:1757-4676
Container-title:EMBO Molecular Medicine
language:en
Short-container-title:EMBO Mol Med

Author:

Maino Eleonora¹²^ORCID,Wojtal Daria¹²,Evagelou Sonia L¹,Farheen Aiman¹,Wong Tatianna W Y¹²,Lindsay Kyle¹,Scott Ori¹³⁴,Rizvi Samar Z¹²,Hyatt Elzbieta¹,Rok Matthew¹²,Visuvanathan Shagana¹,Chiodo Amanda¹,Schneeweiss Michelle¹,Ivakine Evgueni A¹⁵,Cohn Ronald D¹²³⁴^ORCID

Affiliation:

1. Program in Genetics and Genome Biology the Hospital for Sick Children Research Institute Toronto ON Canada

2. Department of Molecular Genetics University of Toronto Toronto ON Canada

3. Institute of Medical Science University of Toronto Toronto ON Canada

4. Department of Pediatrics the Hospital for Sick Children Toronto ON Canada

5. Department of Physiology University of Toronto Toronto ON Canada

Funder

Duchenne Research Fund

Publisher

EMBO

Subject

Molecular Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.15252/emmm.202013228

Reference73 articles.

1. Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense

2. Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy

3. Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

4. A large tandem duplication within the COL4A5 gene is responsible for the high prevalence of Alport syndrome in French Polynesia

5. Increased connective tissue growth factor associated with cardiac fibrosis in the mdx mouse model of dystrophic cardiomyopathy

Cited by 21 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Correction of exon 2, exon 2–9 and exons 8–9 duplications in DMD patient myogenic cells by a single CRISPR/Cas9 system;Scientific Reports;2024-09-11

2. An Irak1-Mecp2 tandem duplication mouse model for the study of MECP2 duplication syndrome;Disease Models & Mechanisms;2024-07-01

3. CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments;Cells;2024-05-08

4. The BALB/c.mdx62 mouse exhibits a dystrophic muscle pathology and is a model of Duchenne muscular dystrophy;Disease Models & Mechanisms;2024-04-01

5. Prevention of early-onset cardiomyopathy in Dmd exon 52–54 deletion mice by CRISPR-Cas9-mediated exon skipping;Molecular Therapy - Methods & Clinical Development;2023-09

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