Foster K, Foster H, Dickson J G
Centre for Biomedical Sciences, School of Biological Sciences, Royal Holloway University of London, Egham, Surrey, UK.
Gene Ther. 2006 Dec;13(24):1677-85. doi: 10.1038/sj.gt.3302877. Epub 2006 Oct 26.
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder affecting 1/3500 male births. There is currently no effective treatment, but gene therapy approaches are offering viable avenues for treatment development. The last 10 years have seen the development of a number of strategies and tools for muscle gene therapy. However, the major hurdle has been the inability to deliver vectors at high enough efficiency via a systemic route. The last 2-3 years (reviewed here) have seen unrivalled progress in efficient systemic delivery of viral and non-viral gene transfer agents and antisense oligonucleotides. This progress, coupled with the successful completion of the first gene therapy clinical trial for DMD, has led to three more clinical trials planned for the immediate future.
杜氏肌营养不良症（DMD）是一种严重的肌肉萎缩性疾病，影响着1/3500的男性新生儿。目前尚无有效的治疗方法，但基因治疗方法为治疗开发提供了可行的途径。在过去十年中，已经开发出了多种用于肌肉基因治疗的策略和工具。然而，主要障碍是无法通过全身途径以足够高的效率递送载体。在过去的2 - 3年（本文对此进行了综述）中，在病毒和非病毒基因转移剂以及反义寡核苷酸的高效全身递送方面取得了无与伦比的进展。这一进展，再加上首个DMD基因治疗临床试验的成功完成，使得在不久的将来又计划开展三项临床试验。
