小干扰RNA在临床治疗中的应用

RNA沉默现象是真核细胞生物一种重要的基因调节机制。利用小干扰RNA影响基因表达的RNA干扰技术在基因功能研究及基因治疗中得到了很大发展。目前已有数种针对不同疾病的siRNA药物进入了临床试验阶段。本文重点介绍了针对呼吸道合胞病毒的小干扰RNA——ALN—RSV01的临床试验进展情况。将小干扰RNA作为分子药物治疗疾病具有广阔的发展空间。但在临床试验及应用中需要以生物医学中的无害、有利、尊重．公正原则进行合理的风险一收益评估．以期其能更好地发挥作用。

Application of siRNA in Clinical Therapy

RNA silencing is an evolutionarily conserved gene-regulatory mechanism that occurs in a wide variety of eukaryotic organisms. Introduce synthetic sequence-specific small interfering RNA （siRNA） to the cell can target and cleave complementary mRNA, It has been reported that synthetic siRNAs are capable of knocking down targets in various diseases in vivo which show their therapeutic potential as small-molecule drugs. Today, there are promising data from ongoing clinical trails for several diseases, this review focus on the clinical trials of respiratory syncytial virus （SRV） specific siRNA-ALN-RSV01, Despite all the progress that have made in the therapeutic use of siRNA, there is the need to perform a riks-benefit analysis based on the four essential ethical principles in biomedicine： nonmaleficence, beneficence, respect for autonomy and justice.