A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy

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  A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy

  By Martin Krahn, Nicolas Wein, Marc Bartoli, William Lostal, Sébastien Courrier, Nathalie Bourg-Alibert, Karine Nguyen, Christophe Vial, Nathalie Streichenberger, Véronique Labelle, Danielle DePetris, Christophe Pécheux, France Leturcq, Pierre Cau, Isabelle Richard, Nicolas Lévy

  Science Translational Medicine22 Sep 2010 : 50ra69

  A 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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  A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy

  By Martin Krahn, Nicolas Wein, Marc Bartoli, William Lostal, Sébastien Courrier, Nathalie Bourg-Alibert, Karine Nguyen, Christophe Vial, Nathalie Streichenberger, Véronique Labelle, Danielle DePetris, Christophe Pécheux, France Leturcq, Pierre Cau, Isabelle Richard, Nicolas Lévy

  Science Translational Medicine22 Sep 2010 : 50ra69

  A 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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