Brolin Camilla, Shiraishi Takehiko
Center for Experimental Drug and Gene Electrotransfer (CEDGE); Department of Oncology 54B1; Copenhagen University Hospital Herlev, Copenhagen, Denmark.
Artif DNA PNA XNA. 2011 Jan;2(1):6-15. doi: 10.4161/adna.2.1.15425.
Duchenne Muscular Dystrophy (DMD) is a lethal disease caused by mutations in the dystrophin gene (DMD) that result in the absence of essential muscle protein dystrophin. Among many different approaches for DMD treatment, exon skipping, mediated by antisense oligonucleotides, is one of the most promising methods for restoration of dystrophin expression. This approach has been tested extensively targeting different exons in numerous models both in vitro and in vivo. During the past 10 years, there has been a considerable progress by using DMD animal models involving three types of antisense oligonucleotides (2'-O-methyl phosphorothioate (2OME-PS), phosphorodiamidate morpholino oligomer (PMO)) and peptide nucleic acid (PNA).
杜氏肌营养不良症（DMD）是一种由肌营养不良蛋白基因（DMD）突变引起的致命疾病，该突变导致必需的肌肉蛋白肌营养不良蛋白缺失。在DMD治疗的众多不同方法中，由反义寡核苷酸介导的外显子跳跃是恢复肌营养不良蛋白表达最有前景的方法之一。这种方法已在体外和体内的众多模型中针对不同外显子进行了广泛测试。在过去10年中，使用涉及三种反义寡核苷酸（2'-O-甲基硫代磷酸酯（2OME-PS）、吗啉代磷酰胺寡聚物（PMO））和肽核酸（PNA）的DMD动物模型取得了相当大的进展。
