Tel Aviv Medical Center, The Genetic Institute, 6 Weizmann Street, 6423906, Tel Aviv, Israel.
Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
J Mol Neurosci. 2017 Oct;63(2):216-222. doi: 10.1007/s12031-017-0972-3. Epub 2017 Sep 15.
Early-onset Parkinson's disease (EOPD) is less common than the typical adult-onset PD and may be associated with a genetic etiology. Mutations in several genes are known to cause autosomal recessive (AR) PD. This study aimed to detect the etiology of EOPD in consanguineous families or families living in a specific small geographic region in Israel. Six families with EOPD affecting more than a single individual were recruited. Homozygous mapping analysis using a single-nucleotide polymorphism-based array was performed in all families, followed by Sanger sequencing of related genes based on the mapping results. In addition, all families underwent PARK2 sequencing and testing for large deletions and duplications in PD-associated genes. Different truncating mutations were detected in the PARK2 gene among affected individuals of three families: c.996C>A (p.Cys332X) and c.101delA in either homozygous or compound heterozygous fashion. Exon 4 deletion was detected in a heterozygous manner in a late-onset PD and in homozygous state in early-onset disease in the same family. No disease-causing mutations were detected in any other tested genes. In total, mutations in the PARK2 gene were detected in four of the six tested families with a history of EOPD. These results further demonstrate the role of PARK2 in AR PD. We recommend genetic analysis for the PARK2 gene when AR PD is suspected.
早发性帕金森病 (EOPD) 比典型的成人发病型帕金森病少见,可能与遗传病因有关。几个基因的突变已知会导致常染色体隐性 (AR) PD。本研究旨在检测以色列一个特定小地理区域内的近亲家庭或家庭中 EOPD 的病因。招募了六家受 EOPD 影响的多个人的家庭。对所有家庭进行了基于单核苷酸多态性的阵列的纯合子映射分析,然后根据映射结果对相关基因进行 Sanger 测序。此外,所有家庭都进行了 PARK2 测序,并对 PD 相关基因中的大片段缺失和重复进行了检测。在三个家庭的受影响个体中检测到 PARK2 基因中的不同截断突变:c.996C>A (p.Cys332X) 和 c.101delA 以纯合或复合杂合方式。在同一家庭中,外显子 4 缺失以杂合方式存在于迟发性 PD 中,以纯合状态存在于早发性疾病中。在任何其他测试的基因中均未检测到致病突变。总共,在六家具有 EOPD 病史的测试家庭中,有四家检测到 PARK2 基因的突变。这些结果进一步证明了 PARK2 在 AR PD 中的作用。当怀疑 AR PD 时,我们建议进行 PARK2 基因的遗传分析。