Rodriguez-Lebron E, Paulson H L
Department of Neurology, Carver College of Medicine, Iowa City, IA 52242, USA.
Gene Ther. 2006 Mar;13(6):576-81. doi: 10.1038/sj.gt.3302702.
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.
通过RNA干扰抑制毒性基因的表达为人类疾病的治疗带来了巨大希望。等位基因特异性方法现已用于沉默与几种神经疾病相关的显性毒性基因。在此，我们根据RNA干扰生物学领域的最新进展，综述用于实现等位基因特异性沉默的策略。特别是，对小干扰RNA和微小RNA加工的新见解可用于提高RNA干扰疗法的效率和特异性。我们还将进一步讨论为最大化这项强大技术的治疗益处可采取的步骤。
