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This volume explores the latest advancements in the field of RNA therapeutics, with a particular focus on the development of exon skipping and inclusion therapies as targeted treatments for genetic diseases, as well as the evaluation methods used post-treatment. The chapters in this book cover both established methods and novel techniques used my researchers working on developing therapies that have proven to be promising for several diseases, including Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and other genetic disorders characterized by abnormal splicing events. 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.
Cutting-edge and thorough, Splice Switching: Methods and Protocols is a valuable resource for researchers across various fields. This book will help in the advancement of basic RNA biology research and aid in accelerating therapeutic developments.
Published by: Humana
Publication Date: 2025-10-01
Format: Hardcover
ISBN-13: 9781071647370
DOI: 10.1007/978-1-0716-4738-7
Dimensions: 254cm x178cm
Pages: 226