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Although peripheral organs are affected, the progressive neurodegeneration in the brain is typically most deleterious, leading to dystonia, ataxia, seizures, and premature death. Although the two genes underlying this disorder in humans and mouse models of the disease have been identified (NPC1 in 95% and NPC2\/HE1 in 5% of human cases), their cellular roles have not Been fully defined, and there is currently no effective treatment for this disorder. To help address these issues, we constructed a recombinant adenovirus, Ad(NPC1\u2010GFP), which contains a cDNA encoding a mouse NPC1 protein with a green fluorescent protein (GFP) fused to its C\u2010terminus. Fluorescence microscopy and cholesterol trafficking assays demonstrate that the GFP\u2010tagged NPC1 protein is functional and detectable in cells from different species (hamster, mouse, human) and of different types (ovary\u2010derived cells, fibroblasts, astrocytes, neurons from peripheral and central nervous systems) in vitro. Combined with results from time\u2010lapse microscopy and in vivo brain injections, our findings suggest that this adenovirus offers advantages for expressing NPC1 and analyzing its cellular localization, movement, functional properties, and beneficial effects in vitro and in vivo. \u00a9 2005 Wiley\u2010Liss, Inc.<\/jats:p>","DOI":"10.1002\/jnr.20592","type":"journal-article","created":{"date-parts":[[2005,8,11]],"date-time":"2005-08-11T18:18:13Z","timestamp":1123784293000},"page":"706-719","source":"Crossref","is-referenced-by-count":9,"title":["Adenovirus expressing an NPC1\u2010GFP fusion gene corrects neuronal and nonneuronal defects associated with Niemann pick type C 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