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.
| ISBN: | 9781493993543 |
| Publication date: | 10th December 2019 |
| Author: | Toshifumi Yokota, Rika Maruyama |
| Publisher: | Humana Press an imprint of Springer New York |
| Format: | Paperback |
| Pagination: | 569 pages |
| Series: | Methods in Molecular Biology |
| Genres: |
Medical genetics |