Department of Experimental Hematology, Hannover Medical School, OE6960-HBZ, Carl-Neuberg-Strasse 1, D-30625 Hannover, Germany.
Viruses. 2011 Jun;3(6):677-713. doi: 10.3390/v3060677. Epub 2011 Jun 3.
Retroviruses are evolutionary optimized gene carriers that have naturally adapted to their hosts to efficiently deliver their nucleic acids into the target cell chromatin, thereby overcoming natural cellular barriers. Here we will review-starting with a deeper look into retroviral biology-how Murine Leukemia Virus (MLV), a simple gammaretrovirus, can be converted into an efficient vehicle of genetic therapeutics. Furthermore, we will describe how more rational vector backbones can be designed and how these so-called self-inactivating vectors can be pseudotyped and produced. Finally, we will provide an overview on existing clinical trials and how biosafety can be improved.
逆转录病毒是进化优化的基因载体，它们已经自然适应了宿主，能够有效地将其核酸递送到靶细胞染色质中，从而克服了天然的细胞屏障。在这里，我们将回顾——从深入了解逆转录病毒生物学开始——如何将简单的γ逆转录病毒鼠白血病病毒（MLV）转化为有效的基因治疗载体。此外，我们将描述如何设计更合理的载体骨架，以及如何对这些所谓的自我失活载体进行假型化和生产。最后，我们将概述现有的临床试验以及如何提高生物安全性。
