代谢疾病的基因治疗

Chandler Randy J, Venditti Charles P

Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.

Transl Sci Rare Dis. 2016;1(1):73-89. doi: 10.3233/TRD-160007.

Gene therapy has recently shown great promise as an effective treatment for a number of metabolic diseases caused by genetic defects in both animal models and human clinical trials. Most of the current success has been achieved using a viral mediated gene addition approach, but gene-editing technology has progressed rapidly and gene modification is being actively pursued in clinical trials. This review focuses on viral mediated gene addition approaches, because most of the current clinical trials utilize this approach to treat metabolic diseases.

基因治疗最近在动物模型和人类临床试验中，作为治疗多种由基因缺陷引起的代谢疾病的有效方法展现出了巨大潜力。目前的大部分成功是通过病毒介导的基因添加方法实现的，但基因编辑技术发展迅速，基因修饰正在临床试验中积极探索。本综述聚焦于病毒介导的基因添加方法，因为目前大多数临床试验都采用这种方法来治疗代谢疾病。