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Potential targets of gene therapy in the treatment of heart failure

机译:基因治疗治疗心力衰竭的潜在目标

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Introduction: Heart failure (HF) is one of the most prevalent diseases; it affects millions of people every year. In addition to being one of the major causes of mortality, it generates a financial burden on healthcare systems. Despite progress in developing new Pharmaceuticals intended to treat HF, even the newest therapies are not satisfactory. Ischemic heart disease often requires operational treatment which decreases the patient's quality of life. The emergence of gene therapy as a viable treatment option for monogenic disorders has resulted in the approach becoming a topic of study in cardiology. Areas covered: The identification of molecular targets could enable a new form of treatment for HF. This review discusses the current advances related to the implementation of gene therapy for those genes connected to the regulation of calcium concentrations. Several trials have recently investigated the efficacy of gene therapy in HF treatment. Researchers have identified SERCA2a, S100A1 and IPP-1 as potential therapeutic targets. Another therapeutic approach could relate to the gene expression regulatory process called SUMOylation.Commentary: Researchers still face a long road ahead; however, overcoming several significant problems will likely lead to a greatly improved prognosis in many patients.
机译:简介:心力衰竭(HF)是最普遍的疾病之一;它每年都会影响数百万人。除了成为死亡率的主要原因之一外,它还为医疗保健系统产生了金融负担。尽管在开发旨在治疗HF的新药物方面取得进展,即使最新的疗法也不令人满意。缺血性心脏病通常需要运营治疗,这会降低患者的生活质量。基因治疗作为单一的单一疾病的可行治疗选择的出现导致了该方法成为心脏病学研究的主题。覆盖区域:分子靶标的鉴定可以为HF进行新的治疗形式。本综述讨论了与将基因治疗实施相关的当前进展,这些基因与钙浓度的调节有关。最近有几种试验研究了基因治疗在HF治疗中的疗效。研究人员已经确定了Serca2a,s100a1和IPP-1作为潜在的治疗目标。另一种治疗方法可以涉及称为Sumoylation的基因表达调控过程:研究人员仍然面临着漫长的道路;然而,克服了几种重大问题可能导致许多患者的预后大大提高。

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