Interdisciplinary program in Genetics, University of Iowa, Iowa City, IA 52242, USA.
Neurotherapeutics. 2013 Jul;10(3):473-85. doi: 10.1007/s13311-013-0183-8.
Over the last decade, RNA interference technology has shown therapeutic promise in rodent models of dominantly inherited brain diseases, including those caused by polyglutamine repeat expansions in the coding region of the affected gene. For some of these diseases, proof-of concept studies in model organisms have transitioned to safety testing in larger animal models, such as the nonhuman primate. Here, we review recent progress on RNA interference-based therapies in various model systems. We also highlight outstanding questions or concerns that have emerged as a result of an improved (and ever advancing) understanding of the technologies employed.
在过去的十年中，RNA 干扰技术在显性遗传性脑疾病的啮齿动物模型中显示出了治疗潜力，包括那些由受影响基因编码区的多聚谷氨酰胺重复扩展引起的疾病。对于其中一些疾病，在模式生物中进行的概念验证研究已经过渡到更大动物模型（如非人类灵长类动物）的安全性测试。在这里，我们回顾了各种模型系统中基于 RNA 干扰的治疗方法的最新进展。我们还强调了由于对所使用技术的理解（以及不断提高）而出现的一些悬而未决的问题或关注点。
