Ribaï Pascale, Depienne Christel, Fedirko Estelle, Jothy Anne-Catherine, Viveweger Caterine, Hahn-Barma Valérie, Brice Alexis, Durr Alexandra
Department of Genetics and Cytogenetics, AP-HP, Groupe Hospitalier Pitié-Salpêtrière, Paris, France.
Eur J Hum Genet. 2008 Jan;16(1):97-104. doi: 10.1038/sj.ejhg.5201922. Epub 2007 Oct 24.
Mutations and deletions in the SPG4 gene are responsible for up to 40% of autosomal dominant hereditary spastic paraplegia (HSP). Patients have pyramidal signs in the lower limbs and some present additional features including cognitive impairment such as executive dysfunction or subcortical dementia. We report 13 patients from three SPG4 families, who had spastic paraplegia associated with mental retardation (n=1), extensive social dependence (n=10), or isolated psychomotor delay (n=2). In family FSP-698, 10 affected individuals had both HSP and mental deficiency leading to social dependence in 9 and institutionalization in 5. The mean age at onset of spastic paraplegia was 11+/-20 years, ranging from 1 to 51 years. This phenotype segregated either with a novel p.Glu442Lys mutation or the two previously described p.Arg459Thr and p.Arg499Cys substitutions in the SPG4 gene. Since two of these mutations were previously reported in families with a pure form of the disease, another genetic factor linked to SPG4 could be responsible for this complex phenotype.
SPG4基因的突变和缺失导致高达40%的常染色体显性遗传性痉挛性截瘫(HSP)。患者下肢有锥体束征,部分患者还有其他特征,包括认知障碍,如执行功能障碍或皮质下痴呆。我们报告了来自三个SPG4家族的13名患者,他们患有与智力迟钝(n = 1)、广泛社会依赖(n = 10)或孤立的精神运动发育迟缓(n = 2)相关的痉挛性截瘫。在FSP - 698家族中,10名受累个体同时患有HSP和智力缺陷,其中9人导致社会依赖,5人需要机构照料。痉挛性截瘫的平均发病年龄为11±20岁,范围从1岁到51岁。这种表型与SPG4基因中的一种新的p.Glu442Lys突变或之前描述的两种p.Arg459Thr和p.Arg499Cys替代突变相关。由于之前在纯合形式疾病的家族中报告过其中两种突变,另一个与SPG4相关的遗传因素可能导致了这种复杂的表型。