In this study, a rAAV vector carrying a reporter gene,Infusion of approximately 6 × 10 9 particles (2 × 10 5 infec-'humanized' green fluorescent protein (GFP), linked to the tious units) of rAAV-MBP-GFP into mouse brains resulted transcriptional control region from the myelin basic protein in the GFP expression specifically in white matter. The (MBP) gene (a myelin-forming cell-specific gene) was con-GFP protein was detected 15 days later by immunostainstructed. Transduction of oligodendrocytes was carried out ing, specifically in the oligodendrocytes. No astrocytes both in vitro and in vivo. The GFP expression was detected were transduced. Our studies suggested that cell types for at least 3 weeks in both transduced oligodendrocyte cell other than neurons in the central nervous system can also line (MOCH-1 cells) and primary cultures of rat oligodendbe transduced by rAAV using a cell-type-specific transcriprocytes. Preferential GFP expression in oligodendrocytes tional control region or promoter. The MBP transcriptional was observed in the primary cultures. In contrast, transduccontrol region might be suitable for gene therapy and other tion with rAAV carrying the CMV promoter produced neurobiology studies requiring direct targeting to the myelistronger GFP fluorescence in various cell types, with the nating cells. majority of GFP-expressing cells being the astrocytes.
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