心力衰竭的基因治疗

Manuel Lobo González
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引用次数: 0

摘要

心力衰竭患者的预期寿命较短,生活质量差,经常住院,尽管在过去几年中已经开发了专门的护理单位,新的药物和设备。因此,需要新的策略来治疗日益增多的心力衰竭患者。对心力衰竭疾病中涉及的许多基本分子机制的更好理解,使得使用不同载体的基因治疗模型得以发展,包括专门针对这些分子靶点的病毒载体。尽管动物研究取得了令人鼓舞的结果,但随机临床试验的临床转化并不成功。本文简要综述了基因转移治疗的基本原理及其在心力衰竭领域的应用,以及最近在心力衰竭患者中进行的试验。
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Terapia génica en insuficiencia cardiaca

Patients with heart failure still have a reduced life expectancy, poor quality of life and frequent hospital admissions, although specific care units, new drugs and devices have been developed in the last years. For that reason, novel strategies are needed to treat the growing population of heart failure patients. Better understanding of many of the basic molecular mechanisms involved in the heart failure disease has allowed the development of gene therapy models using different vectors, including viral vectors specifically directed to these molecular targets. In spite of the promising results from animal studies, the clinical translation by randomized clinical trials has been unsuccessful. This review briefly reviews the principles underlying gene transfer therapy and its application in the field of heart failure, as well as the recent trials carried out in patients with heart failure.

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