Rescue of a Dystrophin-like Protein by Exon Skipping In Vivo Restores GABAA-receptor Clustering in the Hippocampus of the mdx Mouse

Dystrophin, the cytoskeletal protein whose defect is responsible for Duchenne muscular dystrophy (DMD), is normally expressed in both muscles and brain. Genetic loss of brain dystrophin in the mdx mouse model of DMD reduces the capacity for type A γ-aminobutyric acid (GABAA)-receptor clustering in c...

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Veröffentlicht in:	Molecular therapy 2010-09, Vol.18 (9), p.1683-1688
Hauptverfasser:	Vaillend, Cyrille, Perronnet, Caroline, Ros, Carine, Gruszczynski, Carole, Goyenvalle, Aurélie, Laroche, Serge, Danos, Olivier, Garcia, Luis, Peltekian, Elise
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Blotting, Western Brain Cognitive Sciences Dystrophin Dystrophin - genetics Dystrophin - metabolism Exons Exons - genetics Female Hippocampus Hippocampus - metabolism Immunohistochemistry Life Sciences Male Mice Mice, Inbred C57BL Mice, Inbred mdx Muscular dystrophy Muscular Dystrophy, Duchenne Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - therapy Musculoskeletal system Mutation Neurobiology Neurons and Cognition Neurosciences Original Polymerase Chain Reaction Proteins Psychology and behavior Receptors, GABA-A Receptors, GABA-A - genetics Receptors, GABA-A - metabolism
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