RNA干扰技术治疗疾病

RNA Interference Therapy

RNA干扰(RNA interference,RNAi)现象最早发现于秀丽隐杆线虫(Caenorhabditis elegans),随后发现该现象普遍存在于真菌、植物和哺乳动物等真核生物,并行使基因调控和抵御外源基因片段侵袭的作用。目前,RNAi分子机制和RNAi在基因功能方面的研究已经取得了突破性的进展。鉴于RNAi在基因沉默中的特异性、高效性和易操作,其在药物筛选和疾病治疗等方面有着广泛的应用前景。然而,RNAi技术用于治疗疾病的安全性尚待确定,分子传递途径也有待进一步的研究。

RNA interference （RNAi） was discovered in Caenorhabditis elegans firstly, then it was considered existing widely on eukaryotes （including fungi, plant and mammalian） and playing a role in gene regulation and cellular defense. At present, the breakthrough has achieved in RNAi molecular mechanism and gene function. In view of the sequence-specificity, effectivity and simplicity of the technology, RNAi has widespread application prospect in drug screening and therapy. However, it will be important to assess the safety of the RNAi-based therapies, and the further research on the systemic delivery of the siRNA should be done.