Abstract
Complete deficiency of HPRT results in a devastating neurological disease, Lesch-Nyhan syndrome. We have constructed two vectors derived from HSV-1 containing expressible human HPRT cDNA, designated HSV-HP40 and HSV-HP87. HPRT deficient cultured rat neuronal cells infected with these vectors express human HPRT at levels comparable to those in wild type rat neuronal cells. Stable transformation to the HPRT+ phenotype was observed In 10−3 to 10−4 of infected cells. These vectors were slightly less cytopathic than wild-type HSV-1 (strain KOS). We are attempting to reduce viral cytopathic effect by UV-irradiation of the recombinant virions which renders HSV-1 replication defective while maintaining transforming capacity. These experiments demonstrate the feasibility of HSV-1 mediated HPRT gene transfer into neuronal cells. In vivo infection of mice is currently in progress.
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Palella, T., Silverman, L., Levine, M. et al. 107 HERPES SIMPLEX VIRUS (HSV-1) MEDIATED HUMAN HYPOXANTHINE-GUANINE PHOSPHORIBOSYLTRANSFERASE (HPRT) GENE TRANSFER INTO NEURONAL CELLS. Pediatr Res 24, 129 (1988). https://doi.org/10.1203/00006450-198807000-00131
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DOI: https://doi.org/10.1203/00006450-198807000-00131