Molecular Medicine Laboratory, International Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
J Control Release. 2012 Jul 20;161(2):377-88. doi: 10.1016/j.jconrel.2012.04.008. Epub 2012 Apr 10.
After over 20 years from the first application of gene transfer in humans, gene therapy is now a mature discipline, which has progressively overcome several of the hurdles that prevented clinical success in the early stages of application. So far, the vast majority of gene therapy clinical trials have exploited viral vectors as very efficient nucleic acid delivery vehicles both in vivo and ex vivo. Here we summarize the current status of viral gene transfer for clinical applications, with special emphasis on the molecular properties of the major classes of viral vectors and the information so far obtained from gene therapy clinical trials.
自首次将基因转移应用于人类以来,已经过去了 20 多年,基因治疗现在已经发展成为一门成熟的学科,它逐渐克服了早期应用中阻碍临床成功的几个障碍。到目前为止,绝大多数基因治疗临床试验都利用病毒载体作为体内和体外非常有效的核酸传递载体。在这里,我们总结了病毒基因转移用于临床应用的现状,特别强调了主要类型的病毒载体的分子特性以及从基因治疗临床试验中获得的信息。