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杜氏肌营养不良症的治疗研究进展
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Abstract:
杜氏肌营养不良症(Duchenne muscular dystrophy, DMD)是一种x连锁的进行性致死性神经肌肉疾病，这种疾病无法治愈，预期寿命在30岁至50岁之间，死亡与心脏或呼吸并发症有关。近年来，DMD的治疗也取得了一些重要进展，极具前景的CRISPR/cas9介导的基因组编辑有望从基因水平彻底治愈DMD，本文将对DMD治疗的研究现状进行综述，并分析不同治疗策略在其治疗中的研究进展和潜力。
Duchenne muscular dystrophy (Duchenne muscular dystrophy, DMD) is a kind of x-linked progressive fatal neuromuscular disease, the disease cannot be cured, life expectancy is between the ages of 30 to 50, and death is associated with heart or respiratory complications. In recent years, the treatment of DMD has also made some important progress. The promising CRISPR/Cas9-mediated genome editing is expected to completely cure DMD from the gene level. This paper will review the current research status of DMD treatment and analyze the research progress and potential of different treatment strategies in its treatment.
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