Guetta Esther, Peleg Leah
Danek Gertner Institute of Human Genetics, Chaim Sheba Medical Center, Tel-Hashomer, Israel.
Methods Mol Biol. 2008;444:147-59. doi: 10.1007/978-1-59745-066-9_11.
Tay-Sachs disease is an autosomal recessive storage disease caused by the impaired activity of the lysosomal enzyme hexosaminidase A. In this fatal disease, the sphingolipid GM2 ganglioside accumulates in the neurons. Due to high carrier rates and the severity of the disease, population screening and prenatal diagnosis of Tay-Sachs disease are routinely carried out in Israel. Laboratory diagnosis of Tay-Sachs is carried out with biochemical and DNA-based methods in peripheral and umbilical cord blood, amniotic fluid, and chorionic villi samples. The assay of hexosaminidase A (Hex A) activity is carried out with synthetic substrates, 4-methylumbelliferyl-6-sulfo-N-acetyl-beta-glucosaminide (4-MUGS) and 4-methylumbelliferil-N-acetyl-beta-glucosamine (4-MUG), and the DNA-based analysis involves testing for the presence of specific known mutations in the alpha-subunit gene of Hex A. Prenatal diagnosis of Tay-Sachs disease is accomplished within 24-48 h from sampling. The preferred strategy is to simultaneously carry out enzymatic analysis in the amniotic fluid supernatant or in chorionic villi and molecular DNA-based testing in an amniotic fluid cell-pellet or in chorionic villi.
泰-萨克斯病是一种常染色体隐性遗传性贮积病,由溶酶体酶己糖胺酶A活性受损引起。在这种致命疾病中,鞘脂GM2神经节苷脂在神经元中蓄积。由于携带率高且疾病严重,以色列常规开展泰-萨克斯病的群体筛查和产前诊断。泰-萨克斯病的实验室诊断采用外周血和脐带血、羊水及绒毛膜绒毛样本的生化检测和基于DNA的检测方法。己糖胺酶A(Hex A)活性检测采用合成底物4-甲基伞形酮基-6-磺基-N-乙酰-β-氨基葡萄糖苷(4-MUGS)和4-甲基伞形酮基-N-乙酰-β-氨基葡萄糖(4-MUG),基于DNA的分析则是检测己糖胺酶Aα亚基基因中特定已知突变的存在情况。泰-萨克斯病的产前诊断在采样后24至48小时内完成。首选策略是同时对羊水上清液或绒毛膜绒毛进行酶分析,并对羊水细胞沉淀或绒毛膜绒毛进行基于DNA分子的检测。