¹Department of Neurology, Carver College of Medicine, Iowa City, IA, USA

Correspondence: Dr E Rodriguez-Lebron, EMRB Room 240A, Iowa City, IA 52242, USA. E-mail: edgardo-rodriguez@uiowa.edu

Received 30 August 2005; Revised 1 November 2005; Accepted 14 November 2005; Published online 15 December 2005.

Abstract

Suppressing the expression of toxic genes through RNAi holds great promise for the treatment of human disease. Allele-specific approaches have now been used to silence dominant toxic genes implicated in several neurological disorders. Here, we review strategies used to achieve allele-specific silencing in light of recent developments in the field of RNAi biology. In particular, new insights into siRNA and miRNA processing may be used to improve efficiency and specificity of RNAi therapy. We further discuss steps that can be taken to maximize the therapeutic benefits of this powerful technology.