载体介导杜氏肌营养不良症的基因治疗研究现状

杜氏肌营养不良症(DMD)是由肌营养不良蛋白缺失或减少引起的全身肌肉渐进性损伤和运动功能减退的致死性基因遗传病。目前关于DMD患者的治疗手段越来越多,包括药物治疗、物理治疗、基因治疗、干细胞疗法和运动训练疗法等。本文梳理了近20年来载体介导DMD基因治疗情况,发现使用短序列肌营养不良蛋白基因在治疗DMD模型小鼠和犬类身上具有一定效果,并得到一些有效的实验数据证明载体介导基因治疗可以改善动物肌肉形态和功能。但人体临床试验还需要进一步考察载体介导DMD基因治疗的安全性和有效性。

Research status of vector-mediated gene therapy for Duchenne muscular dystrophy

Duchenne muscular dystrophy is a fatal genetic disease with gradual damage of muscles of the whole body and the degradation of motor functions caused by the loss or reduction of dystrophin.Currently,there are more and more treatments for DMD patients,including drug therapy,physical therapy,gene therapy,stem cell therapy and exercise training therapy.After reviewing vector-mediated DMD gene therapy in the past 20 years,the author finds that the use of micro-dystrophin gene has certain effect in the treatment of DMD model mice and dogs,and obtains some effective experimental data to prove that vector-mediated gene therapy can improve the muscle morphology and function of animals.However,the safety and efficacy of vector-mediated DMD gene therapy need to be further investigated in human clinical trials.