Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse-Reference-Cited by-同舟云学术

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

Published:2022-09 Issue: Volume:26 Page:279-293
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Wein Nicolas,Vetter Tatyana A.,Vulin Adeline,Simmons Tabatha R.,Frair Emma C.,Bradley Adrienne J.,Gushchina Liubov V.,Almeida Camila F.,Huang Nianyuan,Lesman Daniel,Rajakumar Dhanarajan,Weiss Robert B.,Flanigan Kevin M.

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference44 articles.

1. Evidence-based path to newborn screening for Duchenne muscular dystrophy;Mendell;Ann. Neurol.,2012

2. Improvement of survival in Duchenne Muscular Dystrophy: retrospective analysis of 835 patients;Passamano;Acta Myol.,2012

3. The pathology of paralysis with muscular degeneration (paralysie myosclerotique), or paralysis with apparent hypertrophy;Duchenne;Br. Med. J.,1867

4. DMD mutations in 576 dystrophinopathy families: a step forward in genotype-phenotype correlations;Juan-Mateu;PLoS One,2015

5. Dystrophin protects the sarcolemma from stresses developed during muscle contraction;Petrof;Proc. Natl. Acad. Sci. USA,1993

Cited by 20 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. Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping;Molecular Therapy - Nucleic Acids;2024-09

2. Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study;Journal of Neuromuscular Diseases;2024-04-30

3. Molecular mechanisms and therapeutic strategies for neuromuscular diseases;Cellular and Molecular Life Sciences;2024-04-28

4. Adeno-associated virus as a delivery vector for gene therapy of human diseases;Signal Transduction and Targeted Therapy;2024-04-03

5. CRISPR-Based Gene Editing Techniques in Pediatric Neurological Disorders;Pediatric Neurology;2024-04