假性肥大型肌营养不良的治疗进展  

Advance in therapy for Duchenne/Becker muscular dystrophy

作　　者：许田田 蓝丹[1] 

机构地区：[1]广西医科大学第一附属医院儿科,广西南宁530021

出　　处：《中国当代儿科杂志》2015年第3期294-298,共5页Chinese Journal of Contemporary Pediatrics

摘　　要：假性肥大型进行性肌营养不良是一类最常见的X连锁隐性遗传性肌肉疾病,进行性肌无力是主要的临床特征,抗肌萎缩蛋白基因的突变是致病原因。治疗方法一直是研究热点,近年来随着分子生物学诊断治疗技术的日臻成熟,假性肥大型进行性肌营养不良的治疗也有新的突破,该文从传统疗法、基因疗法、细胞移植疗法等方面总结其治疗进展。Duchenne/Becker muscular dystrophy （DMD/BMD） is the most common X-linked recessive inherited neuromuscular disease, characterized by progressive muscle weakness. Mutations in the dystrophin gene are responsible for this disease. Treatment for this disease has always been a topic of interest. With the development of diagnosis and treatment technology of molecular biology, promising therapies have been developed. This review article summarizes the advance in traditional therapy, cell transplantation and gene therapy for this disease.

关 键 词：假性肥大型肌营养不良 基因疗法 细胞移植 治疗 

分 类 号：R746.2[医药卫生—神经病学与精神病学]