Work

Alternative Splicing as a Therapeutic Target for Human Diseases

Year: 2009

Type: book-chapter

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.

Source: Methods in molecular biology

DOI: 10.1007/978-1-60327-295-7_10

Authors: Kenneth J. Dery, Veronica Gusti, Shikha Gaur, John E. Shively, Yun Yen, Rajesh K. Gaur

Institution: Beckman Research Institute

Language: English

Topic: RNA Research and Splicing

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: green

Funder: National Cancer Institute