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肥厚型心肌病是单基因常染色体显性遗传性疾病,是导致青少年猝死的常见原因.传统治疗多以改善症状为主,无法延缓心肌肥厚乃至心力衰竭的进展.近年来,随着分子生物学技术的飞速发展,基因治疗在传统治疗的基础上为肥厚型心肌病临床治疗提供了新思路.现就肥厚型心肌病的基因治疗研究进展进行综述.“,”Hypertrophic cardiomyopathy is a monogenic heart disease and the most frequent cause of sudden death in young people. Gene therapy to treat hypertrophic cardiomyopathy is an appealing strategy because of the limited therapeutic options available to manage the most severe hypertrophic cardiomyopathy.Nevertheless,the growing understanding of the molecular basis of these diseases,and the development of sophisticated viral vectors and delivery strategies are providing researchers with adequate means to target specific genes and pathways involved in disorders of hypertrophic cardiomyopathy.Data from preclinical studies have demonstrated that gene therapy can be successfully used to relief or prevent hypertrophic cardiomyopathy.Therefore,gene therapy might plausibly become a treatment option for patients with difficulty to manage inherited hypertrophic cardiomyopathy.In this review,we summarize the preclinical studies into gene therapy for hypertrophic cardiomyopathy.