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Gene therapy for cardiovascular disease: Perspectives and potential

机译:心血管疾病的基因治疗:前景和潜力

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摘要

Cardiovascular disease is the most frequent cause of mortality in the western world, accounting for over 800,000 premature deaths per year in the EU alone. Cardiovascular disease is the second most common application for gene therapy clinical trials, which most frequently employ adenovirus serotype 5 (Ad5)-based vectors as delivery vehicles. Although interactions of Ad5 vectors with circulating proteins and cells can limit their efficacy after systemic administration, local gene delivery strategies show great potential in the cardiovascular setting, notably in the context of vascular delivery. Here we review the pathogenesis of bypass graft failure and in-stent restenosis, identifying potential therapeutic targets and discussing recent advances in the field of adenovirus biology and retargeting that, in concert, will potentially translate in coming years to more effective gene therapies for cardiovascular applications.
机译:在西方世界,心血管疾病是最常见的死亡原因,仅在欧盟,每年就有80万例过早死亡。心血管疾病是基因治疗临床试验中第二常见的应用,该试验最频繁地使用基于腺病毒血清型5(Ad5)的载体作为载体。尽管全身给药后Ad5载体与循环蛋白和细胞的相互作用可能会限制其功效,但局部基因递送策略在心血管环境中显示出巨大潜力,尤其是在血管递送的情况下。在这里,我们回顾了旁路移植失败和支架内再狭窄的发病机制,确定了潜在的治疗靶标,并讨论了腺病毒生物学领域的最新进展,并将其重新定位为未来几年可能转化为心血管应用更有效的基因疗法的目标。 。

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