Günzburg W H, Salmons B
GSF-Forschungszentrum für Umwelt und Gesundheit, Institut für Molekulare Virologie, Oberschleissheim, Germany.
J Mol Med (Berl). 1996 Apr;74(4):171-82. doi: 10.1007/BF00204747.
The transfer of genes of potential therapeutic benefit is presently being attempted in the clinic to treat a number of genetic and virally induced diseases. Many of these protocols use retroviral vectors derived from murine leukemia retroviruses as gene delivery systems. Although these viral delivery systems are well suited for this purpose, a number of their characteristics, some of which are discussed here, are still troublesome. Future retroviral vectors will incorporate nonretroviral features and will be tailored to desired needs for specific uses. These vectors will be safer, more efficient, and targeted in their delivery. Further, expression of the therapeutic genes carried will be limited to the specific target cell type. Some of the recent advances that have been made towards this goal are reviewed here.
目前正在临床中尝试转移具有潜在治疗益处的基因,以治疗多种遗传和病毒诱导的疾病。许多这些方案使用源自鼠白血病逆转录病毒的逆转录病毒载体作为基因递送系统。尽管这些病毒递送系统非常适合此目的,但它们的一些特性(这里讨论了其中一些)仍然存在问题。未来的逆转录病毒载体将纳入非逆转录病毒特征,并将根据特定用途的期望需求进行定制。这些载体将更安全、更高效,并且在递送方面具有靶向性。此外,所携带的治疗性基因的表达将仅限于特定的靶细胞类型。本文综述了为实现这一目标所取得的一些最新进展。