Major T, Svetel M, Romac S, Kostić V S
PCR Center, Faculty of Biology, University of Belgrade, Yugoslavia.
J Neurol. 2001 Nov;248(11):940-3. doi: 10.1007/s004150170045.
Primary torsion dystonia (PTD) is a clinically and genetically heterogeneous movement disorder. A GAG deletion at position 946 in the DYT1 gene is responsible for most cases of autosomal dominant early-onset PTD. We analysed the DYT1 mutation in 50 patients from a Serbian population, selected according to the proposed guidelines for diagnostic testing: (a) 38 patients with PTD onset < 26 years, and (b) 12 patients with the disease onset +/- 26 years, but with at least one affected family member with early-onset dystonia. Only three apparently sporadic patients among the 50 individuals tested were positive for the GAG deletion in the DYT1 gene: one with typical, generalized, one with long-lasting, non-progressive segmental, and one with multifocal dystonia. Molecular analysis of relatives in 2 families revealed that the lack of family history was due to reduced penetrance.
原发性扭转性肌张力障碍(PTD)是一种临床和遗传异质性的运动障碍。DYT1基因第946位的GAG缺失是大多数常染色体显性早发性PTD病例的病因。我们根据诊断测试的建议指南,对来自塞尔维亚人群的50名患者的DYT1突变进行了分析:(a)38例PTD发病年龄小于26岁的患者,以及(b)12例发病年龄为±26岁,但至少有一名早发性肌张力障碍的患病家庭成员的患者。在接受检测的50人中,只有3名明显散发的患者DYT1基因的GAG缺失呈阳性:一名患有典型全身性肌张力障碍,一名患有长期非进行性节段性肌张力障碍,一名患有多灶性肌张力障碍。对2个家族亲属的分子分析表明,家族史的缺失是由于外显率降低所致。