Lehn P M
BMT Unit, Hospital Saint-Louis, Paris, France.
Bone Marrow Transplant. 1990 May;5(5):287-93.
The use of recombinant retroviruses for gene transfer into the haematopoietic tissue in vivo is still a new area of research. The initial results of in vitro studies were very exciting. In contrast, the early in vivo studies in mice were somewhat disappointing because of the transient and low levels of expression of the transferred gene. Recently, however, better results have been obtained in the murine system in vivo. New packaging cell lines have been constructed, which are safer and still efficient. Better vectors have been designed. Thus, significant levels of expression of the transgene have been achieved in murine long-term transplant recipients. However, the results obtained so far in large animal studies are still disappointing. It should be emphasized that further progress must be based on a simple overall strategy involving better understanding of the functioning of the gene to be transferred by gene expression studies, design of vectors carrying a fully active and correctly regulated minilocus and better knowledge of the biological properties of the target cells, the haematopoietic stem cells.
将重组逆转录病毒用于体内造血组织的基因转移仍是一个新的研究领域。体外研究的初步结果非常令人振奋。相比之下，早期在小鼠体内进行的研究结果却有些令人失望，因为转移基因的表达是短暂且水平较低的。然而，最近在小鼠体内系统中已取得了更好的结果。构建了新的包装细胞系，这些细胞系更安全且仍然高效。设计了更好的载体。因此，在小鼠长期移植受体中已实现了转基因的显著表达水平。然而，迄今为止在大型动物研究中获得的结果仍然令人失望。应该强调的是，进一步的进展必须基于一个简单的总体策略，包括通过基因表达研究更好地理解待转移基因的功能、设计携带完全活性且调控正确的小基因座的载体以及更深入了解靶细胞即造血干细胞的生物学特性。
