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Recent findings into the potential of gene therapy to reverse heart failure.

机译:基因治疗逆转心力衰竭潜力的最新发现。

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The incidence of heart failure (HF) is ever growing and the mortality of HF patients is similar to patients suffering from cancer disease. The central clinical problem is a lack of therapies to target the underlying molecular defects that lead to chronic ventricular dysfunction. Substantial evidence points to a final common pathway in failing myocardium, including distinct changes in intracellular Ca2+-cycling and beta-adrenergic receptor signaling. An attractive strategy to address these alterations is cardiac gene therapy and several distinct approaches have been undertaken during the last decade with impressing therapeutic benefit, at least in animal HF models. The present focus of research is the clinical translation of cardiac gene therapy including the optimization of vectors, delivery strategies and testing the compatibility with established pharmacologic treatment to improve the prognosis of HF in the near future.
机译:心力衰竭(HF)的发病率不断增长,HF患者的死亡率与罹患癌症的患者相似。中心临床问题是缺乏针对潜在导致慢性心室功能不全的潜在分子缺陷的疗法。大量证据指出了心肌衰竭的最终共同途径,包括细胞内Ca2 +循环和β-肾上腺素能受体信号转导的明显变化。解决这些改变的一种有吸引力的策略是心脏基因治疗,并且在过去的十年中,至少在动物HF模型中,已经采取了几种具有令人印象深刻的治疗效果的独特方法。目前的研究重点是心脏基因治疗的临床翻译,包括载体的优化,递送策略以及测试与已确立的药物治疗的相容性以改善HF的预后。

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