Zimran A, Sorge J, Gross E, Kubitz M, West C, Beutler E
Department of Molecular and Experimental Medicine, Scripps Clinic and Research Foundation, La Jolla, California 92037.
J Clin Invest. 1990 Jan;85(1):219-22. doi: 10.1172/JCI114415.
The molecular diagnosis of Gaucher disease has been difficult due to the existence of several different point mutations in the glucocerebrosidase gene and due to the presence of a tightly linked, highly homologous pseudogene. We now report the occurrence of a "Lepore-like" glucocerebrosidase fusion gene in which the 5' end is the functional gene and the 3' end is the pseudogene. This further complicates the molecular diagnosis of Gaucher disease but sheds light on the molecular anatomy of the glucocerebrosidase gene complex and on the pathogenesis of this important storage disease.
由于葡萄糖脑苷脂酶基因中存在几种不同的点突变,并且存在紧密连锁、高度同源的假基因,戈谢病的分子诊断一直很困难。我们现在报告一种“类Lepore”葡萄糖脑苷脂酶融合基因的出现,其中5'端是功能基因,3'端是假基因。这进一步使戈谢病的分子诊断复杂化,但为葡萄糖脑苷脂酶基因复合体的分子结构以及这种重要贮积病的发病机制提供了线索。