Smaili Imane, Hajjaj Imane, Razine Rachid, Tibar Houyam, Salmi Ayyoub, Bouslam Naima, Moussa Ahmed, Regragui Wafa, Bouhouche Ahmed
Research Team in Neurology and Neurogenetics, Genomics Center of Human Pathologies, Faculty of Medicine and Pharmacy, University Mohammed V, Rabat, Morocco.
Department of Neurology, Hassan II Regional Hospital Center, Agadir, Morocco.
Case Rep Genet. 2020 Dec 3;2020:8813344. doi: 10.1155/2020/8813344. eCollection 2020.
Parkinson's disease (PD) is the second most common neurodegenerative disorder after Alzheimer disease. Five to ten percent of patients have monogenic form of the disease, while most of sporadic PD cases are caused by the combination of genetic and environmental factors. Microtubule-associated protein tau (MAPT) has been appointed as one of the most important risk factors for several neurodegenerative diseases including PD. MAPT is characterized by an inversion in chromosome 17 resulting in two distinct haplotypes H1 and H2. Studies described a significant association of MAPT H1j subhaplotype with PD risk, while H2 haplotype was associated with Parkinsonism, particularly to its bradykinetic component. We report here an isolated case displaying an akinetic-rigid form of PD, with age of onset of 41 years and a good response to levodopa, who developed dementia gradually during the seven years of disease progression. The patient does not carry the LRRK2 G2019S mutation, copy number variations, nor pathogenic and rare variants in known genes associated with PD. MAPT subhaplotype genotyping revealed that the patient has the H1j/H2 diplotype, his mother H1j/H1j, his two healthy brothers H1j/H1v and his deceased father was by deduction H1v/H2. The H1j/H2 diplotype was shown in a total of 3 PD patients among 80, who also did not have known PD-causing mutation and in 1 out of 92 healthy individual controls. The three patients with this diplotype all have a similar clinical phenotype. Our results suggest that haplotypes H1j and H2 are strong risk factor alleles, and their combination could be responsible for early onset of PD with dementia.
帕金森病(PD)是仅次于阿尔茨海默病的第二常见神经退行性疾病。5%至10%的患者患有单基因形式的该疾病,而大多数散发性PD病例是由遗传和环境因素共同导致的。微管相关蛋白tau(MAPT)已被确定为包括PD在内的几种神经退行性疾病的最重要风险因素之一。MAPT的特征是17号染色体发生倒位,产生两种不同的单倍型H1和H2。研究表明,MAPT H1j亚单倍型与PD风险显著相关,而H2单倍型与帕金森综合征相关,尤其是与运动迟缓成分相关。我们在此报告一例孤立病例,该病例表现为运动不能-强直型PD,发病年龄为41岁,对左旋多巴反应良好,在疾病进展的七年中逐渐发展为痴呆。该患者未携带LRRK2 G2019S突变、拷贝数变异,也未在与PD相关的已知基因中携带致病和罕见变异。MAPT亚单倍型基因分型显示,该患者具有H1j/H2双倍型,其母亲为H1j/H1j,其两个健康兄弟为H1j/H1v,其已故父亲经推断为H1v/H2。在80例PD患者中,共有3例显示出H1j/H2双倍型,这些患者也没有已知的导致PD的突变,在92例健康个体对照中有1例显示出该双倍型。具有这种双倍型的三名患者都具有相似的临床表型。我们的结果表明,单倍型H1j和H2是强烈的风险因素等位基因,它们的组合可能是导致伴有痴呆的早发性PD的原因。