Gene Therapy for X-Linked Myotubular Myopathy
Author:
Publisher
Springer International Publishing
Link
http://link.springer.com/content/pdf/10.1007/978-3-030-03095-7_32
Reference58 articles.
1. Kumar SR, Markusic DM, Biswas M, High KA, Herzog RW (2016) Clinical development of gene therapy: results and lessons from recent successes. Mol Ther Methods Clin Dev 3:16034
2. Pacak CA, Conlon T, Mah CS, Byrne BJ (2008) Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle. Genet Vaccines Ther 6:14
3. Le Hir M, Goyenvalle A, Peccate C, Précigout G, Davies KE, Voit T, Garcia L, Lorain S (2013) AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy. Mol Ther 21:1551–1558
4. Dupont J-B, Tournaire B, Georger C et al (2015) Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA. Mol Ther Methods Clin Dev 2:15010
5. Buj-Bello A, Fougerousse F, Schwab Y et al (2008) AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis. Hum Mol Genet 17:2132–2143
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