A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy
2010; American Association for the Advancement of Science; Volume: 2; Issue: 50 Linguagem: Inglês
10.1126/scitranslmed.3000951
ISSN1946-6242
AutoresMartin Krahn, Nicolas Wein, Marc Bartoli, William Lostal, Sébastien Courrier, Nathalie Bourg-Alibert, Karine Nguyen, Christophe Vial, Nathalie Streichenberger, Véronique Labelle, D. Depétris, Christophe Pécheux, France Leturcq, Pierre Cau, Isabelle Richard, Nicolas Lévy,
Tópico(s)RNA regulation and disease
ResumoA naturally occurring miniversion of the dysferlin protein found in a patient shows that gene therapy by minigene transfer may be possible in dysferlinopathies.
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