Dandana A, Ferchichi S, Ben Khelifa S, Jaidane Z, Monastiri K, Chkioua L, Maire I, Froissart R, Bonnet V, Laradi S, Miled A
Service de biochimie, CHU Farhat Hached, 4000 Sousse, Tunisie.
Pathol Biol (Paris). 2008 Mar;56(2):88-93. doi: 10.1016/j.patbio.2007.09.029. Epub 2008 Feb 21.
Gaucher disease is one of the most prevalent lysosomal disorders. In this present study, we report a diagnostic strategy of type 1 Gaucher disease. The application of combined methods in molecular biology allowed us to analyse the p.Asn 370 Ser mutation. The affected individual activity is very low. First, we have to used the enzymatic digestion method. Then, we have to identified the mutation by the refractory mutation system technique using specific primers for the p.Asn 370 Ser mutation. These analyses are supplemented by the direct sequencing in order to seek and confirm this mutation. Finally, the absence of the 55 pb deletion in exon 9 among corroborated the presence of the homozygous genotype of this p.Asn 370 Ser in the patient DNA.
戈谢病是最常见的溶酶体贮积症之一。在本研究中,我们报告了1型戈谢病的诊断策略。分子生物学联合方法的应用使我们能够分析p.Asn 370 Ser突变。受影响个体的活性非常低。首先,我们必须使用酶切方法。然后,我们必须使用针对p.Asn 370 Ser突变的特异性引物,通过难熔性突变系统技术鉴定该突变。这些分析通过直接测序进行补充,以寻找和确认该突变。最后,患者DNA中外显子9中55 pb缺失的不存在证实了该p.Asn 370 Ser纯合基因型的存在。