1区 · 医学
Article
作者: DePetris, Danielle ; Krahn, Martin ; Richard, Isabelle ; Lévy, Nicolas ; Cau, Pierre ; Labelle, Véronique ; Wein, Nicolas ; Courrier, Sébastien ; Nguyen, Karine ; Vial, Christophe ; Pécheux, Christophe ; Bourg-Alibert, Nathalie ; Bartoli, Marc ; Lostal, William ; Leturcq, France ; Streichenberger, Nathalie
A naturally occurring miniversion of the dysferlin protein found in a patient shows that gene therapy by minigene transfer may be possible in dysferlinopathies.