Rescue of a dystrophin-like protein by exon skipping normalizes synaptic plasticity in the hippocampus of the mdx mouse

► Loss of brain dystrophin alters GABAr clustering, plasticity and cognitive functions. ► Rescue of brain dystrophin by exon skipping restores GABAr clustering. ► We show that exon skipping normalizes synaptic plasticity in mice lacking dystrophin. ► Exon skipping may thus improve both muscle and brain defects in muscular dystrophy. ► Dystrophin may modulate synaptic plasticity through GABAr clustering in adult brain.