Palella T D, Silverman L J, Schroll C T, Homa F L, Levine M, Kelley W N
Department of Internal Medicine, University of Michigan Medical School, Ann Arbor 48109.
Mol Cell Biol. 1988 Jan;8(1):457-60. doi: 10.1128/mcb.8.1.457-460.1988.
The virtually complete deficiency of the purine salvage enzyme hypoxanthine-guanine phosphoribosyltransferase (HPRT) results in a devastating neurological disease, Lesch-Nyhan syndrome. Transfer of the HPRT gene into fibroblasts and lymphoblasts in vitro and into hematopoietic cells in vivo has been accomplished by other groups with retroviral-derived vectors. It appears to be necessary, however, to transfer the HPRT gene into neuronal cells to correct the neurological dysfunction of this disorder. The neurotropic virus herpes simplex virus type 1 has features that make it suitable for use as a vector to transfer the HPRT gene into neuronal tissue. This report describes the isolation of an HPRT-deficient rat neuroma cell line, designated B103-4C, and the construction of a recombinant herpes simplex virus type 1 that contained human HPRT cDNA. These recombinant viruses were used to infect B103-4C cells. Infected cells expressed HPRT activity which was human in origin.
嘌呤补救酶次黄嘌呤 - 鸟嘌呤磷酸核糖转移酶（HPRT）几乎完全缺乏会导致一种毁灭性的神经系统疾病——莱施 - 奈恩综合征。其他研究小组已通过逆转录病毒衍生载体在体外将HPRT基因导入成纤维细胞和淋巴细胞，在体内将其导入造血细胞。然而，将HPRT基因导入神经细胞似乎对于纠正该疾病的神经功能障碍是必要的。嗜神经病毒单纯疱疹病毒1型具有一些特性，使其适合用作将HPRT基因导入神经组织的载体。本报告描述了一种HPRT缺陷型大鼠神经瘤细胞系（命名为B103 - 4C）的分离，以及一种包含人HPRT cDNA的重组单纯疱疹病毒1型的构建。这些重组病毒被用于感染B103 - 4C细胞。被感染的细胞表达了源自人类的HPRT活性。
