RNA Splicing and Disease: Animal Models to Therapies.

RNA Splicing and Disease: Animal Models to Therapies.
Trends Genet. 2018 Nov 19;:
Authors: Montes M, Sanford BL, Comiskey DF, Chandler DS
Abstract
Alternative splicing of pre-mRNA increases genetic diversity, and recent studies estimate that most human multiexon genes are alternatively spliced. If this process is not highly regulated and accurate, it leads to mis-splicing events, which may result in proteins with altered function. A growing body of work has implicated mis-splicing events in a range of diseases, including cancer, neurodegenerative diseases, and muscular dystrophies. Understanding the mechanisms that cause aberrant splicing events and how this leads to disease is vital for designing effective therapeutic strategies. In this review, we focus on advances in therapies targeting splicing, and highlight the animal models developed to recapitulate disease phenotypes as a model for testing these therapies.
PMID: 30466729 [PubMed - as supplied by publisher]

Source: Trends in Genetics : TIG - November 19, 2018 Category: Genetics & Stem Cells Authors: Montes M, Sanford BL, Comiskey DF, Chandler DS Tags: Trends Genet Source Type: research