Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model-Reference-Cited by-同舟云学术

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Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

Published:2023-12 Issue: Volume:31 Page:101144
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Gushchina Liubov V.,Bradley Adrienne J.,Vetter Tatyana A.,Lay Jacob W.,Rohan Natalie L.,Frair Emma C.,Wein Nicolas,Flanigan Kevin M.

Funder

National Institute of Arthritis and Musculoskeletal and Skin Diseases

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference39 articles.

1. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis;Crisafulli;Orphanet J. Rare Dis.,2020

2. Improved molecular diagnosis of dystrophinopathies in an unselected clinical cohort;Dent;Am. J. Med. Genet.,2005

3. Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule;Aartsma-Rus;Muscle Nerve,2006

4. Duplications in the DMD gene;White;Hum. Mutat.,2006

5. Eteplirsen for the treatment of Duchenne muscular dystrophy;Mendell;Ann. Neurol.,2013

Cited by 2 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. Thrombotic Microangiopathy as an Emerging Complication of Viral Vector–Based Gene Therapy;Kidney International Reports;2024-07

2. Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications;Molecular Therapy - Methods & Clinical Development;2023-12

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