This book presents a comprehensive collection of detailed state-of-the-art exon skipping and splices modulation prools. 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 prools, and tips on troubleshooting and avoiding known pitfalls.
Authoritative and cutting-edge,
Exon Skipping and Inclusion Therapies: Methods and Prools
aims to help researchers initiate the development of next-generation therapies.