Miller R G, Hoffman E P
Department of Neurology, California Pacific Medical Center, San Francisco.
Neurol Clin. 1994 Nov;12(4):699-725.
The increased understanding of the genetic basis for DMD and the pathophysiology of the disease have produced improved techniques of patient and carrier diagnosis, genetic counseling, and new approaches to searching for a treatment. The present level of experimentation with both myoblast implantation and gene therapy may make treatment of this inherited, relentlessly progressive muscle disease possible within the next several years.
对杜氏肌营养不良症(DMD)遗传基础及疾病病理生理学的深入了解,带来了患者及携带者诊断、遗传咨询技术的改进,以及寻找治疗方法的新途径。目前对成肌细胞植入和基因治疗的实验水平,可能使在未来几年内治疗这种遗传性、持续进展性肌肉疾病成为可能。