Adenovirus-mediated gene transfer to retinal cells was evaluated using the replication-defective recombinant adenovirus vector Ad2/CMVlacZ-1 (coding for /I-galactosidase) both in an in vitro murine culture model and in vivo in adult mice. In vitro, no difference in infectability of neuronal and glial cells was observed, and 5096 of neurons expressed the exogenous gene at low viral concentration (10 pfulcell). In vivo, intraocular injection of 3 x IO6 pfu Ad2/CMVlacZ-1 resulted in expression of the transferred /3galactosidase gene in retinal pigment epithelium and ganglion cells. These results demonstrate that Ad2lCMVkzcZ-1 is an effective vector for gene transfer into retinal cells. &y wor& Defective recombinant adenovirus; /I-Galactosidase; Retina; Gene transfer; Retinal cell culture
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