Electrophysiological Evaluation in mdx52 Mouse Brain After Antisense-Mediated Exon 51 or 53 Skipping.

Abstract

Duchenne muscular dystrophy (DMD) is a neuromuscular disease characterized by progressive muscle weakness alongside cognitive and behavioral impairments. Notably, the lack of brain-specific Dp140 isoform increases the risk of neurodevelopmental disorders in DMD. We reported abnormal social behavior and the reduced glutamatergic transmission using electrophysiological techniques in DMD model mice lacking Dp140. Also, we proposed that antisense oligonucleotide (ASO) -induced exon 53 skipping therapy is thought to be promising for neurodevelopmental disorders in DMD lacking Dp140. Here, we describe a detailed protocol for the intracerebroventricular injection and electrophysiological evaluation in the brain of DMD model mice following ASO-induced exon 51 or 53 skipping therapy.