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.
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