Repeat expansion diseases: when a good RNA turns bad
Article first published online: 25 MAY 2010
Copyright © 2010 John Wiley & Sons, Ltd.
Wiley Interdisciplinary Reviews: RNA
Volume 1, Issue 1, pages 173–192, July/August 2010
How to Cite
Dickson, A. M. and Wilusz, C. J. (2010), Repeat expansion diseases: when a good RNA turns bad. WIREs RNA, 1: 173–192. doi: 10.1002/wrna.18
- Issue published online: 22 JUN 2010
- Article first published online: 25 MAY 2010
An increasing number of dominantly inherited diseases have now been linked with expansion of short repeats within specific genes. Although some of these expansions affect protein function or result in haploinsufficiency, a significant portion cause pathogenesis through production of toxic RNA molecules that alter cellular metabolism. In this review, we examine the criteria that influence toxicity of these mutant RNAs and discuss new developments in therapeutic approaches. Copyright © 2010 John Wiley & Sons, Ltd.
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