Department of Dermatology and Allergology, Philipps University, Marburg, Germany.
J Dtsch Dermatol Ges. 2010 Aug;8(8):582-91. doi: 10.1111/j.1610-0387.2010.07435.x. Epub 2010 Apr 19.
The success of gene therapy mainly depends on the gene vector (GV) responsible for the efficient transport of genetic information. The qualities of a GV have a profound influence on the method of application, the efficiency of gene transfer in the target tissue, the amount and persistence of gene expression and the potential side effects and safety risks. Clinical gene therapy studies over the past 20 years have contributed to the development and testing of different GV systems, some of which also show great potential for the treatment of skin diseases. In this review the structures, methods of application, characteristics, clinical uses and possibilities for optimization of these GV will be discussed with regard to their cutaneous applications.
基因治疗的成功主要取决于负责有效传递遗传信息的基因载体(GV)。GV 的质量对应用方法、目标组织中的基因转移效率、基因表达的数量和持久性以及潜在的副作用和安全风险有深远的影响。过去 20 年的临床基因治疗研究促进了不同 GV 系统的发展和测试,其中一些系统在治疗皮肤疾病方面也显示出巨大的潜力。在这篇综述中,将讨论这些 GV 在皮肤应用方面的结构、应用方法、特性、临床应用和优化的可能性。