Department of Neurology, University of Yamanashi, Japan.
Department of Pediatrics, University of Yamanashi, Japan.
Intern Med. 2021 Dec 15;60(24):3969-3974. doi: 10.2169/internalmedicine.7463-21. Epub 2021 Jun 12.
We herein describe a Charcot-Marie-Tooth disease (CMT) family with a MFN2 mutation with atypical ocular manifestations. The proband, his mother, his third daughter, and his deceased maternal grandfather all had symptoms of CMT and a visual impairment (either cataracts or severe astigmatism). On whole-exome sequencing for the proband having CMT and congenital cataracts, we identified a c.314C>T (p.Thr105Met) mutation in MFN2, but no mutation in the causative genes associated with cataracts. This missense mutation in MFN2 co-segregated with CMT and the atypical ocular manifestations in this family. The findings of this study might help to expand the clinical phenotype of heterogeneous MFN2-related CMT.
我们在此描述了一个具有 MFN2 突变的腓骨肌萎缩症(CMT)家族,其具有非典型的眼部表现。先证者、其母亲、其三女儿和已故的外祖父均有 CMT 和视力障碍(白内障或严重散光)的症状。在先证者患有 CMT 和先天性白内障的全外显子组测序中,我们在 MFN2 中鉴定出 c.314C>T(p.Thr105Met)突变,但与白内障相关的致病基因中没有突变。MFN2 中的这个错义突变与该家族的 CMT 和非典型眼部表现共分离。这项研究的结果可能有助于扩大异质性 MFN2 相关 CMT 的临床表型。