Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice

• Main Authors: Flavien Bizot (Author), Remko Goossens (Author), Thomas Tensorer (Author), Sergei Dmitriev (Author), Luis Garcia (Author), Annemieke Aartsma-Rus (Author), Pietro Spitali (Author), Aurélie Goyenvalle (Author)
• Format: Book
• Published: Elsevier, 2022-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Antisense-mediated exon skipping is one of the most promising therapeutic strategies for Duchenne muscular dystrophy (DMD), and some antisense oligonucleotide (ASO) drugs have already been approved by the US FDA despite their low efficacy. The potential of this therapy is still limited by several ch...