Cell-based gene therapy for the prevention and treatment of cardiac dysfunction.

Marc S Penn
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引用次数: 21

Abstract

A substantial need exists for new treatments to prevent and treat cardiac dysfunction. In the 1990s, there was great hope for gene therapy in this regard. Since that time, the focus has switched to cell therapy-in particular, therapy-with the aim of inducing myocardial regeneration. Individually, gene and cell therapies still have substantial promise. Ultimately, however, the convergence of both techniques might be necessary to achieve improvements in cardiac function and more successful clinical outcomes in patients with cardiac dysfunction. This approach has already been adopted for treatment of malignancies. Several gene products are currently being studied, including growth factors and chemokines that can modulate the survival and function of cardiac myocytes following an ischemic event and influence remodeling of the left ventricle. However, several issues remain, including the optimization and characterization of cell types, selection of vectors for gene transfer, and identification of appropriate strategies for delivery. Here, we review the potential and need for cell-based gene therapy for the prevention and treatment of cardiac dysfunction and attempt to discuss the unresolved issues.

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以细胞为基础的基因疗法预防和治疗心功能障碍。
迫切需要新的治疗方法来预防和治疗心功能障碍。在20世纪90年代,基因治疗在这方面有很大的希望。从那时起,焦点转向了细胞疗法,特别是以诱导心肌再生为目标的疗法。单独来看,基因和细胞疗法仍有很大的前景。然而,最终,两种技术的融合可能是必要的,以实现心功能的改善和心功能障碍患者更成功的临床结果。这种方法已被用于治疗恶性肿瘤。目前正在研究几种基因产物,包括生长因子和趋化因子,它们可以在缺血事件后调节心肌细胞的存活和功能,并影响左心室的重塑。然而,仍然存在一些问题,包括细胞类型的优化和表征,基因转移载体的选择,以及适当递送策略的确定。在这里,我们回顾了细胞基因治疗在预防和治疗心功能障碍方面的潜力和需求,并试图讨论尚未解决的问题。
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