Rando Thomas A
Department of Neurology and Neurological Sciences, SUMC, Room A-343, Stanford University School of Medicine, Stanford, CA 94305-5235, USA.
Biochim Biophys Acta. 2007 Feb;1772(2):263-71. doi: 10.1016/j.bbadis.2006.07.009. Epub 2006 Jul 28.
Duchenne muscular dystrophy (DMD) is one of the most common lethal, hereditary diseases of childhood. Since the identification of the genetic basis of this disorder, there has been the hope that a cure would be developed in the form of gene therapy. This has yet to be realized, but many different gene therapy approaches have seen dramatic advances in recent years. Although viral-mediated gene therapy has been at the forefront of the field, several non-viral gene therapy approaches have been applied to animal and cellular models of DMD. These include plasmid-mediated gene delivery, antisense-mediated exon skipping, and oligonucleotide-mediated gene editing. In the past several years, non-viral gene therapy has moved from the laboratory to the clinic. Advances in vector design, formulation, and delivery are likely to lead to even more rapid advances in the coming decade. Given the relative simplicity, safety, and cost-effectiveness of these methodologies, non-viral gene therapy continues to have great promise for future gene therapy approaches to the treatment of DMD.
杜氏肌营养不良症（DMD）是儿童期最常见的致死性遗传性疾病之一。自从确定了这种疾病的遗传基础以来，人们一直希望能开发出基因疗法来治愈它。这一目标尚未实现，但近年来许多不同的基因疗法都取得了显著进展。尽管病毒介导的基因疗法一直处于该领域的前沿，但几种非病毒基因疗法已应用于DMD的动物和细胞模型。这些方法包括质粒介导的基因递送、反义介导的外显子跳跃和寡核苷酸介导的基因编辑。在过去几年中，非病毒基因疗法已从实验室走向临床。载体设计、制剂和递送方面的进展可能会在未来十年带来更快的进步。鉴于这些方法相对简单、安全且具有成本效益，非病毒基因疗法在未来治疗DMD的基因疗法中仍具有巨大潜力。
