Morgan J E
Department of Histopathology, Charing Cross and Westminster Medical School, London, UK.
Hum Gene Ther. 1994 Feb;5(2):165-73. doi: 10.1089/hum.1994.5.2-165.
Experiments in mice have supported the idea of treating Duchenne muscular dystrophy (DMD) by implanting normal muscle precursor cells into dystrophin-deficient muscles. However, similar experiments on DMD patients have had little success. Gene therapy for DMD, by introducing dystrophin constructs via retroviral or adenoviral vectors, has been shown to be possible in the mouse, but the efficiency and safety aspects of this technique will have to be carefully examined before similar experiments can be attempted in man. Direct injection of dystrophin cDNA constructs into mdx muscles has given rise to very low levels of dystrophin and this may be a possibility for the treatment of heart muscle.
在小鼠身上进行的实验支持了通过将正常肌肉前体细胞植入缺乏抗肌萎缩蛋白的肌肉中来治疗杜氏肌营养不良症（DMD）的想法。然而，在DMD患者身上进行的类似实验收效甚微。通过逆转录病毒或腺病毒载体引入抗肌萎缩蛋白构建体对DMD进行基因治疗，在小鼠身上已被证明是可行的，但在对人类进行类似实验之前，必须仔细研究该技术的效率和安全性。将抗肌萎缩蛋白cDNA构建体直接注射到mdx小鼠肌肉中，产生的抗肌萎缩蛋白水平非常低，这可能是治疗心肌的一种方法。
