Alternative Splicing as a Therapeutic Target for Human Diseases
Open Access
- 29 May 2009
- book chapter
- research article
- Published by Springer Science and Business Media LLC
- Vol. 555, 127-144
- https://doi.org/10.1007/978-1-60327-295-7_10
Abstract
The majority of eukaryotic genes undergo alternative splicing, an evolutionarily conserved phenomenon, to generate functionally diverse protein isoforms from a single transcript. The fact that defective pre-mRNA splicing can generate non-functional and often toxic proteins with catastrophic effects, accurate removal of introns and joining of exons is vital for cell homeostasis. Thus, molecular tools that could either silence a disease-causing gene or regulate its expression in trans will find many therapeutic applications. Here we present two RNA-based approaches, namely RNAi and theophylline-responsive riboswitch that can regulate gene expression by loss-of-function and modulation of splicing, respectively. These strategies are likely to continue to play an integral role in studying gene function and drug discovery.Keywords
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