Exon Skipping and Inclusion Therapies Methods and Protocols [Hardcover]

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.

Part I: Basics and Introductions

1. Invention and Early History of Exon Skipping and Splice Modulation

Kenji Rowel Q. Lim and Toshifumi Yokota

 

2. An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases

Merryl Rodrigues and Toshifumi Yokota

 

3. Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy

 

4. Nusinersen in the Treatment of Spinal Muscular Atrophy

Kara Goodkey, Tejal Aslesh, Rika Maruyama, and Toshifumi Yokota

 

Part II: Design of AONs and Strategy

 

5. Tips to Design Effective Splice-switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion

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