Zhu B X, Zhang L, Wang H Y, Wang R, Jia W
Shaanxi Eye Hospital, Xi'an People's Hospital (Xi'an Fourth Hospital), Xi'an 710004, China.
Zhonghua Yan Ke Za Zhi. 2024 Feb 11;60(2):180-184. doi: 10.3760/cma.j.cn112142-20231213-00288.
Different from classical autosomal recessive Wolfram syndrome, Wolfram-like syndrome is an autosomal dominant disorder caused by a heterozygous mutation in the WFS1 gene. In this case, a 7-year-old male child presented to the eye clinic due to vision loss that could not be corrected, discovered during a routine examination. The child had experienced hearing impairment since early childhood, leading to cochlear implantation. Ophthalmic examination revealed optic disc atrophy in both eyes. Optical coherence tomography imaging demonstrated a distinctive thickening of the outer plexiform layer with abnormal layering, characteristic of a single mutation in the WFS1 gene. Subsequent genetic testing identified a de novo heterozygous missense mutation c.2051C>T (p.A684V) in the WFS1 gene, which ultimately led to the diagnosis of Wolfram-like syndrome.
与经典的常染色体隐性遗传性沃夫勒姆综合征不同,类沃夫勒姆综合征是一种常染色体显性疾病,由WFS1基因的杂合突变引起。在本病例中,一名7岁男童因视力丧失无法矫正而就诊于眼科诊所,该视力丧失是在常规检查中发现的。该患儿自幼年起就有听力障碍,因此接受了人工耳蜗植入。眼科检查发现双眼视盘萎缩。光学相干断层扫描成像显示外丛状层有明显增厚且分层异常,这是WFS1基因单突变的特征。随后的基因检测在WFS1基因中发现了一个新发的杂合错义突变c.2051C>T(p.A684V),最终确诊为类沃夫勒姆综合征。