Transfer of Human HPRT Gene Sequence into Neuronal Cells by a Herpes Simplex Virus Derived Vector
The purine salvage enzyme, hypoxanthine-guanine phosphoribosyltrans-ferase (HPRT), catalyzes the conversion of hypoxanthine and guanine to the mononucleotides, IMP and GMP, respectively. Complete deficiency of HPRT activity in humans causes the Lesch-Nyhan syndrome, a devastating disease characterized by overproduction of uric acid and severe neurological dysfunction.1, 2 For a variety of reasons, attention has focused on the Lesch-Nyhan syndrome as one of the initial candidates for therapy by somatic cell gene transfer.3
KeywordsThymidine Kinase HPRT Activity Human HPRT APRT Gene Herpes Virus Thymidine Kinase
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