Davidson Beverly L, Paulson Henry L
Department of Internal Medicine, University of Iowa, Iowa City, IA 52242, USA.
Lancet Neurol. 2004 Mar;3(3):145-9. doi: 10.1016/S1474-4422(04)00678-7.
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.
RNA干扰（RNAi）的最新发现彻底改变了生物学研究，如今有望成为治疗人类疾病的一种潜在疗法。目前无法治疗的神经疾病尤其具有吸引力。科学家们已经成功地在体外使用RNAi抑制显性疾病基因；在某些情况下，这种抑制具有等位基因特异性，能使致病等位基因沉默，同时维持正常等位基因的表达。现在的挑战是将这项强大的技术应用于动物模型体内，以抑制疾病基因并纠正疾病表型。在应对这一挑战的过程中，研究应受益于近期在向大脑进行病毒和非病毒治疗递送方面取得的进展。
