Mengel David, Librizzi Damiano, Schoser Benedikt, Gläser Dieter, Clemen Christoph S, Dodel Richard, Schröder Rolf
Klinik für Neurologie, Philipps-Universität Marburg, Marburg.
Klinik für Nuklearmedizin, Philipps-Universität Marburg, Marburg.
Fortschr Neurol Psychiatr. 2018 Jul;86(7):434-438. doi: 10.1055/s-0044-101033. Epub 2018 Jul 20.
Mutations of the human VCP gene, which encodes the V: alosin C: ontaining P: rotein (synonyms: p97, TER ATPase), are associated with various multi-systemic protein aggregation diseases. We report on a patient with progressive myopathy and incipient cognitive deficits. A diagnostic muscle biopsy revealed an inclusion body myopathy with protein aggregates. Magnetic resonance imaging and F18-positron-emission-tomography disclosed a fronto-temporal atrophy and glucose hypometabolism of the frontal and temporal lobes, respectively. Based on the clinical findings, a genetic analysis was performed which revealed a heterozygous c.277C>T (p.Arg93Cys) mutation of the VCP gene, thus confirming the diagnosis of IBMPFD (I: nclusion B: ody M: yopathie with P: aget Disease of the Bones and F: ronto-temporal D: ementia).
人类VCP基因发生突变,该基因编码含缬酪肽蛋白(同义词:p97、TER ATP酶),与多种多系统蛋白聚集疾病相关。我们报告了1例患有进行性肌病和早期认知缺陷的患者。诊断性肌肉活检显示为伴有蛋白聚集体的包涵体肌病。磁共振成像和F18正电子发射断层扫描分别显示额颞叶萎缩和额颞叶葡萄糖代谢减低。基于临床发现进行了基因分析,结果显示VCP基因存在杂合性c.277C>T(p.Arg93Cys)突变,从而确诊为包涵体肌病合并佩吉特骨病和额颞叶痴呆(IBMPFD)。