Hara K, Kashiwamata S, Ogasawara N, Ohishi H, Natsume R, Yamanaka T, Hakamada S, Miyazaki S, Watanabe K
Tohoku J Exp Med. 1982 Jul;137(3):275-82. doi: 10.1620/tjem.137.275.
The classical Lesch-Nyhan syndrome has the deficiency of hypoxanthine-guanine phosphoribosyltransferase (HGPRT) activity as the result of mutation in the structural gene for the enzyme located on the X chromosome and is believed to be of X-linked recessive or sex-linked mode of inheritance. This is the first report of a girl who showed typical clinical features and biochemical characteristics of the classical Lesch-Nyhan syndrome. Her mother was not a heterozygote for a deficiency of HGPRT. Possible genetic mechanisms responsible for this case were discussed.
经典的莱施-奈恩综合征由于位于X染色体上的该酶结构基因突变导致次黄嘌呤-鸟嘌呤磷酸核糖转移酶(HGPRT)活性缺乏,被认为是X连锁隐性或性连锁遗传模式。这是首例表现出经典莱施-奈恩综合征典型临床特征和生化特性的女孩的报告。她的母亲并非HGPRT缺乏的杂合子。文中讨论了该病例可能的遗传机制。