Hatefi Arash, Canine Brenda F
Department of Pharmaceutical Sciences, Center for Integrated Biotechnology, Washington State University, Pullman, WA, USA.
Gene Ther Mol Biol. 2009;13(A):15-19.
Gene therapy is perceived as a revolutionary technology with the promise to cure almost any disease, provided that we understand its genetic basis. However, enthusiasm has rapidly abated as multiple clinical trials have failed to show efficacy. The limiting factor seems to be the lack of a suitable delivery system to carry the therapeutic genes to the target tissue safely and efficiently. Therefore, advancements in cancer gene therapy in general depend on the development of novel vectors with maximum therapeutic efficacy at the target site and minimal toxicity to normal tissues. This mini-review highlights both the major fortes and the unique challenges associated with the state-of-the-art gene carriers currently being used in cancer gene therapy.
基因治疗被视为一项革命性技术，有望治愈几乎任何疾病，前提是我们了解其遗传基础。然而，随着多项临床试验未能显示出疗效，人们的热情迅速消退。限制因素似乎是缺乏一种合适的递送系统，能够将治疗性基因安全、高效地输送到靶组织。因此，癌症基因治疗的进展总体上取决于新型载体的开发，这种载体在靶位点具有最大的治疗效果，而对正常组织的毒性最小。这篇小型综述突出了目前癌症基因治疗中所使用的最先进基因载体的主要优势以及独特挑战。
