Exon Skipping and Inclusion Therapies

This fully updated edition presents a collection of protocols reflecting the latest advancements in exon skipping and inclusion strategies. The book explores the design of antisense oligonucleotide therapies, in vitro and in vivo evaluation of exon skipping in Duchenne muscular dystrophy, enhancing exon skipping efficiency, as well as methods involving spinal muscular atrophy and other diseases. W...