Progress in transduction of cerebellar Purkinje cells in vivo using viral vectors.

Expression of a foreign gene in cerebellar Purkinje cells in vivo is a powerful method for exploring the pathophysiology of the cerebellum. Although using developmental engineering many gene-modified mice have been generated, this approach is time-consuming and requires a lot of effort for crossing different lines of mice, genotyping and maintenance of animals. If a gene of interest can be transferred to and efficiently expressed in Purkinje cells of developing and mature animals, it saves much time, effort and money. Recent advances in viral vectors have markedly contributed to selective and efficient gene transfer to Purkinje cells in vivo. There are two approaches for selective gene expression in Purkinje cells: one is to take advantage of the viral tropism for Purkinje cells, which includes the tropism of adeno-associated virus and the vesicular stomatitis virus glycoprotein (VSV-G)-pseudotyped lentivirus. Another method, which might be used in combination with the first one, is utilization of a Purkinje-cell-specific promoter. Focusing mainly on these points, recent progress in viral-vector-mediated transduction of Purkinje cells in vivo is reviewed.