Rajput Alex, Ross Jay P, Bernales Cecily Q, Rayaprolu Sruti, Soto-Ortolaza Alexandra I, Ross Owen A, van Gerpen Jay, Uitti Ryan J, Wszolek Zbigniew K, Rajput Ali H, Vilariño-Güell Carles
Division of Neurology, University of Saskatchewan and Saskatoon Health Region, Saskatoon, SK, Canada.
Department of Medical Genetics, University of British Columbia, Vancouver, BC, Canada.
Eur J Hum Genet. 2015 Jun;23(6):887-8. doi: 10.1038/ejhg.2014.164. Epub 2014 Aug 13.
Exome-sequencing analyses have identified vacuolar protein sorting 35 homolog (VPS35) and DnaJ (Hsp40) homolog, subfamily C, member 13 (DNAJC13) harboring disease-causing variants for Parkinson disease (PD). Owing to the suggested clinical, pathological and genetic overlap between PD and essential tremor (ET) we assessed the presence of two VPS35 and DNAJC13 disease-causing variants in ET patients. TaqMan probes were used to genotype VPS35 c.1858G>A (p.(D620N)) (rs188286943) and DNAJC13 c.2564A>G (p.(N855S)) (rs387907571) in 571 ET patients of European descent, and microsatellite markers were used to define the disease haplotype in variant carriers. Genotyping of DNAJC13 identified two ET patients harboring the c.2564A>G (p.(N855S)) variant previously identified in PD patients. Both patients appear to share the disease haplotype previously reported. ET patients with the VPS35 c.1858G>A (p.(D620N)) variants were not observed. Although a genetic link between PD and ET has been suggested, DNAJC13 c.2564A>G (p.(N855S)) represents the first disease-causing variant identified in both, and suggests the regulation of clathrin dynamics and endosomal trafficking in the pathophysiology of a subset of ET patients.
外显子组测序分析已鉴定出液泡蛋白分选35同源物(VPS35)和DnaJ(Hsp40)同源物C亚家族成员13(DNAJC13)携带帕金森病(PD)的致病变体。鉴于PD和特发性震颤(ET)之间存在临床、病理和遗传重叠,我们评估了ET患者中两种VPS35和DNAJC13致病变体的存在情况。使用TaqMan探针在571名欧洲血统的ET患者中对VPS35 c.1858G>A(p.(D620N))(rs188286943)和DNAJC13 c.2564A>G(p.(N855S))(rs387907571)进行基因分型,并使用微卫星标记来确定变体携带者中的疾病单倍型。DNAJC13的基因分型鉴定出两名ET患者携带先前在PD患者中鉴定出的c.2564A>G(p.(N855S))变体。两名患者似乎共享先前报道的疾病单倍型。未观察到携带VPS35 c.1858G>A(p.(D620N))变体的ET患者。尽管有人提出PD和ET之间存在遗传联系,但DNAJC13 c.2564A>G(p.(N855S))是在两者中首次鉴定出的致病变体,提示网格蛋白动力学和内体运输在一部分ET患者病理生理学中的调控作用。