Li Chengwen, Bowles Dawn E, van Dyke Terry, Samulski Richard Jude
Gene Therapy Center, University of North Carolina (UNC) at Chapel Hill, Chapel Hill, NC 27599, USA.
Cancer Gene Ther. 2005 Dec;12(12):913-25. doi: 10.1038/sj.cgt.7700876.
Augmenting cancer treatment by protein and gene delivery continues to gain momentum based on success in animal models. The primary hurdle of fully exploiting the arsenal of molecular targets and therapeutic transgenes continues to be efficient delivery. Vectors based on adeno-associated virus (AAV) are of particular interest as they are capable of inducing transgene expression in a broad range of tissues for a relatively long time without stimulation of a cell-mediated immune response. Perhaps the most important attribute of AAV vectors is their safety profile in phase I clinical trials ranging from CF to Parkinson's disease. The utility of AAV vectors as a gene delivery agent in cancer therapy is showing promise in preclinical studies. In this review, we will focus on the basic biology of AAV as well as recent progress in the use of this vector in cancer gene therapy.
基于在动物模型中的成功，通过蛋白质和基因递送增强癌症治疗的方法正不断获得发展动力。充分利用分子靶点和治疗性转基因武器库的主要障碍仍然是高效递送。基于腺相关病毒（AAV）的载体特别受关注，因为它们能够在相当长的时间内在广泛的组织中诱导转基因表达，而不会刺激细胞介导的免疫反应。AAV载体最重要的特性或许是其在从囊性纤维化到帕金森病的I期临床试验中的安全性。AAV载体作为癌症治疗中基因递送剂的效用在临床前研究中显示出前景。在这篇综述中，我们将聚焦于AAV的基础生物学以及该载体在癌症基因治疗中的最新进展。
