Raemdonck Koen, Vandenbroucke Roosmarijn E, Demeester Joseph, Sanders Niek N, De Smedt Stefaan C
Laboratory of General Biochemistry and Physical Pharmacy, Faculty of Pharmaceutical Sciences, Ghent University, Harelbekestraat 72, B-9000 Ghent, Belgium.
Drug Discov Today. 2008 Nov;13(21-22):917-31. doi: 10.1016/j.drudis.2008.06.008. Epub 2008 Aug 7.
Since the demonstration of RNA interference (RNAi) in mammalian cells, considerable research and financial effort has gone towards implementing RNAi as a viable therapeutic platform. RNAi is, without doubt, the most promising strategy for the treatment of human genetic disorders. Because many of the targets proposed for RNAi therapy require chronic treatment, researchers agree that the emphasis must now be placed on the safe and long-term application of RNAi drugs to reap the benefits at last.
自从在哺乳动物细胞中证实RNA干扰(RNAi)以来,已经投入了大量的研究和资金,致力于将RNAi作为一个可行的治疗平台。毫无疑问,RNAi是治疗人类遗传疾病最有前景的策略。由于许多提议用于RNAi治疗的靶点都需要长期治疗,研究人员一致认为,现在必须将重点放在RNAi药物的安全和长期应用上,以便最终获得益处。