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. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step and readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and comprehensive, Exon Skipping and Inclusion Therapies: Methods and Protocols, Second Edition serves as a valuable resource for scientists working to refine RNA-targeted therapeutics and translate them into clinical applications.