PDF(Pubmed)
Abstract：
The primary etiology of a diverse range of cardiomyopathies is now understood to be genetic, creating a new paradigm for targeting treatments on the basis of the underlying molecular cause. This review provides a genetic and etiologic context for the traditional clinical classifications of cardiomyopathy, including molecular subtypes that may exhibit differential responses to existing or emerging treatments. The authors describe several emerging cardiomyopathy treatments, including gene therapy, direct targeting of myofilament function, protein quality control, metabolism, and others. The authors discuss advantages and disadvantages of these approaches and indicate areas of high potential for short- and longer term efficacy.
摘要：
各种心肌病的主要病因现在被认为是遗传的，在潜在分子原因的基础上创造一种新的靶向治疗模式。这篇综述为心肌病的传统临床分类提供了遗传和病因学背景，包括可能对现有或新兴治疗表现出不同反应的分子亚型。作者描述了几种新兴的心肌病治疗方法，包括基因疗法,直接靶向肌丝功能，蛋白质质量控制，新陈代谢，和其他人。作者讨论了这些方法的优缺点，并指出了短期和长期疗效的高潜力领域。
