Antisense approaches for the treatment of Duchenne muscular dystrophy and recent advances to address DMD brain comorbidities

This presentation will cover the development of exon-skipping approaches for the treatment of Duchenne muscular dystrophy (DMD) and the recent possibilities to address DMD brain comorbidities. In addition to the progressive muscle weakness and degeneration which are characteristic of DMD, 50% of affected individuals have debilitating central nervous system (CNS) comorbidities, including intellectual disability, neurodevelopmental problems encompassing autism, Attention Deficit Hyperactivity Disorder and Obsessive-Compulsive Disorder. Several antisense tools including antisense oligonucleotides (ASO) and vectorized strategies (AAV-U7) have shown their therapeutic potential for DMD. Can these exon-skipping tools also address the CNS deficits associated with the lack of dystrophin in the brain?