new discovery - RNA interference for neurological disease

[Guest guest]

Molecular medicine for the brain: Silencing of disease genes with RNA

interference

Summary from The Lancet, March 1, 2004

Beverly L son a and Henry L son b

Summary

The recent discovery of RNA interference (RNAi) has revolutionised

biological research and now holds promise as a potential therapy for

human diseases. Currently untreatable neurological diseases are

especially attractive targets. Scientists have already succeeded in

using RNAi to suppress dominant disease genes in vitro; in some cases,

this suppression has been allele-specific, silencing the disease-causing

allele while maintaining expression of the normal allele. The challenge

now is to bring this powerful technology in vivo to animal models to

suppress disease genes and correct disease phenotypes. In the

confrontation of this challenge, research should benefit from recent

advances in viral and non-viral delivery of therapy to the brain.

Affiliations:

a Departments of Internal Medicine, Neurology, and Physiology &

Biophysics, University of Iowa, Iowa City, IA, USA. b Department of

Neurology, University of Iowa, Iowa City, IA, USA.