Wood Matthew J A, Trülzsch Barbara, Abdelgany Amr, Beeson David
Department of Human Anatomy and Genetics, Oxford University, Oxford, UK.
Hum Mol Genet. 2003 Oct 15;12 Spec No 2:R279-84. doi: 10.1093/hmg/ddg275. Epub 2003 Aug 19.
Progress in the understanding of RNA biology has brought into focus the prospect of using RNA-based therapeutics as a novel approach to treat human disease. In particular, following the discovery of the RNA interference (RNAi) pathway, the emergence of technology based on small interfering RNA (siRNA) now offers a powerful and highly specific tool for therapeutic gene silencing. Many neurological diseases, including neurodegenerative disorders, tumours and retinal disease are likely candidates to benefit from such advances. The challenges ahead will be to identify appropriate disease gene targets and, crucially, to understand the biological parameters that determine safe, precise and effective delivery and function of RNA-based therapeutic molecules within the unique environment of the nervous system.
对RNA生物学理解的进展使基于RNA的疗法作为治疗人类疾病的新方法这一前景成为焦点。特别是，随着RNA干扰（RNAi）途径的发现，基于小干扰RNA（siRNA）的技术的出现为治疗性基因沉默提供了一种强大且高度特异性的工具。许多神经疾病，包括神经退行性疾病、肿瘤和视网膜疾病，都可能从这些进展中受益。未来的挑战将是确定合适的疾病基因靶点，至关重要的是，要了解在神经系统独特环境中决定基于RNA的治疗分子安全、精确和有效递送及功能的生物学参数。
