This book presents a comprehensive collection of detailed state-of-the-art exon skipping and
splices modulation protocols. Chapters detail 14 genetic diseases AON-mediated therapies and
CRISPR Cas9-mediated gene editing therapies. Written in the highly successful Methods in
Molecular Biology series format chapters include introductions to their respective topics
lists of the necessary materials and reagents step-by-step readily reproducible laboratory
protocols and tips on troubleshooting and avoiding known pitfalls.Authoritative and
cutting-edge Exon Skipping and Inclusion Therapies: Methods and Protocols aims to help
researchers initiate the development of next-generation therapies.
