Manchester Academic Health Science Centre, School of Biomedicine, Vascular Gene Therapy Unit, Core Technology Facility, The University of Manchester, 46 Grafton Street, Manchester M13 9NT, UK.
Cardiovasc Res. 2011 Sep 1;91(4):565-76. doi: 10.1093/cvr/cvr197. Epub 2011 Jul 8.
Gene transfer within the cardiovascular system was first demonstrated in 1989 yet, despite extensive basic-science and clinical research, unequivocal benefit in the clinical setting remains to be demonstrated. Potential reasons for this include the fact that recombinant viral vectors, used in the majority of clinical studies, have inherent problems with immunogenicity that are difficult to circumvent. Attention has turned therefore to plasmid vectors, which possess many advantages over viruses in terms of safety and ease of use, and many clinical studies have now been performed using non-viral technology. This review will provide an overview of clinical trials for cardiovascular disease using plasmid vectors, recent developments in plasmid delivery and design, and potential directions for this modality of gene therapy.
心血管系统内的基因转移最早于 1989 年得到证实,尽管基础科学和临床研究广泛开展,但在临床环境中明确获益仍有待证实。其潜在原因包括这样一个事实,即大多数临床研究中使用的重组病毒载体存在免疫原性方面的固有问题,难以回避。因此,人们将注意力转向质粒载体,质粒载体在安全性和易用性方面比病毒具有许多优势,并且现在已经使用非病毒技术进行了许多临床研究。这篇综述将概述使用质粒载体治疗心血管疾病的临床试验、质粒传递和设计的最新进展,以及这种基因治疗方式的潜在方向。