摘要
杜兴肌肉萎缩症(DMD)是由抗肌萎缩(dystrophin)蛋白基因发生突变导致的一种进行性、致死性的疾病,患者多为男性,治疗过程涉及多学科、多专业。目前糖皮质激素治疗是应用最广泛的治疗方法,但该方法并不能恢复肌肉中dystrophin蛋白表达,因此治疗效果有限。近年来,随着治疗技术的发展,基因治疗取得了丰硕的成果,是该病目前唯一有效的根治方法,并且已有新药上市。本文对糖皮质类激素治疗和基因治疗的研究进展等加以阐述。
Duchenne muscular dystrophy is a progressive,fatal disease caused by mutations in the dystrophin gene,which needs multidisciplinary treatment. Most patients are male. Glucocorticoid therapy is the most widely used measure,but this method does not restore the expression of dystrophin protein in the muscle,so the treatment effect is limited. In recent years,with the development of therapeutic technology,gene therapy has achieved fruitful results and it is the only effective cure method,and new drugs have already been marketed. This article elaborated on the research progress in glucocorticoid therapy and gene therapy of Duchenne muscular dystrophy.
作者
卜鑫珏
奚鑫
刘松青
杜倩
BU Xin-jue;XI Xin;LIU Song-qing;DU Qian(The Third Affiliated Hospital of Chongqing Medical University(Gener Hospital),Chongqing 401120,China)
出处
《中国新药杂志》
CAS
CSCD
北大核心
2020年第2期165-169,共5页
Chinese Journal of New Drugs
