Vinagre-Aragón Ana, Campo-Caballero David, Mondragón-Rezola Elisabet, Pardina-Vilella Lara, Hernandez Eguiazu Haizea, Gorostidi Ana, Croitoru Ioana, Bergareche Alberto, Ruiz-Martinez Javier
Department of Neurology, Hospital Universitario Donostia, San Sebastián, Spain.
Neuroscience Area, Biodonostia Health Research Institute, San Sebastián, Spain.
Front Neurol. 2021 Mar 2;12:635396. doi: 10.3389/fneur.2021.635396. eCollection 2021.
Parkinson's disease (PD) is characterized by a great clinical heterogeneity. Nevertheless, the biological drivers of this heterogeneity have not been completely elucidated and are likely to be complex, arising from interactions between genetic, epigenetic, and environmental factors. Despite this heterogeneity, the clinical patterns of monogenic forms of PD have usually maintained a good clinical correlation with each mutation once a sufficient number of patients have been studied. Mutations in LRRK2 are the most commonly known genetic cause of autosomal dominant PD known to date. Furthermore, recent genome-wide association studies have revealed variations in LRRK2 as significant risk factors also for the development of sporadic PD. The LRRK2-R1441G mutation is especially frequent in the population of Basque ascent based on a possible founder effect, being responsible for almost 50% of cases of familial PD in our region, with a high penetrance. Curiously, Lewy bodies, considered the neuropathological hallmark of PD, are absent in a significant subset of LRRK2-PD cases. Indeed, these cases appear to be associated with a less aggressive primarily pure motor phenotype. The aim of our research is to examine the clinical phenotype of R1441G-PD patients, more homogeneous when we compare it with sporadic PD patients or with patients carrying other LRRK2 mutations, and reflect on the value of the observed correlation in the genetic forms of PD. The clinical heterogeneity of PD leads us to think that there may be as many different diseases as the number of people affected. Undoubtedly, genetics constitutes a relevant key player, as it may significantly influence the phenotype, with differences according to the mutation within the same gene, and not only in familial PD but also in sporadic forms. Thus, extending our knowledge regarding genetic forms of PD implies an expansion of knowledge regarding sporadic forms, and this may be relevant due to the future therapeutic implications of all forms of PD.
帕金森病(PD)具有显著的临床异质性。然而,这种异质性的生物学驱动因素尚未完全阐明,可能很复杂,是由遗传、表观遗传和环境因素之间的相互作用引起的。尽管存在这种异质性,但一旦研究了足够数量的患者,单基因形式的帕金森病的临床模式通常与每种突变保持良好的临床相关性。LRRK2突变是迄今为止已知的常染色体显性帕金森病最常见的遗传原因。此外,最近的全基因组关联研究表明,LRRK2的变异也是散发性帕金森病发展的重要风险因素。基于可能的奠基者效应,LRRK2-R1441G突变在巴斯克血统人群中尤为常见,在我们地区几乎占家族性帕金森病病例的50%,且具有高外显率。奇怪的是,在LRRK2帕金森病病例的一个重要亚组中不存在被认为是帕金森病神经病理学标志的路易小体。事实上,这些病例似乎与侵袭性较小的主要为纯运动型表型相关。我们研究的目的是检查R1441G帕金森病患者的临床表型,与散发性帕金森病患者或携带其他LRRK2突变的患者相比,该表型更为一致,并思考在帕金森病遗传形式中观察到的相关性的价值。帕金森病的临床异质性使我们认为,受影响的人数有多少,可能就有多少种不同的疾病。毫无疑问,遗传学是一个相关的关键因素,因为它可能会显著影响表型,同一基因内的突变会导致差异,不仅在家族性帕金森病中如此,在散发性形式中也是如此。因此,扩展我们对帕金森病遗传形式的认识意味着对散发性形式的认识的扩展,由于帕金森病所有形式未来的治疗意义,这可能是相关的。