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一位患有线粒体疾病的患者,其病因是 MRPS22 中的一个新突变。

A patient with mitochondrial disorder due to a novel mutation in MRPS22.

机构信息

Sami Ulus Children Hospital, Metabolism Unit, Babur cad. No: 44, 06080, Altındağ, Ankara, Turkey.

Deparment of Radiology, Hacettepe University, Ankara, Turkey.

出版信息

Metab Brain Dis. 2017 Oct;32(5):1389-1393. doi: 10.1007/s11011-017-0074-5. Epub 2017 Jul 27.

Abstract

MRPS22 gene defect is a very rare newly discovered mitochondrial disorder. We report a 4-month-old severely affected male infant with MRPS22 mutation. Whole exome sequencing revealed a novel homozygous splicing mutation c.339 + 5 G > A in MRPS22 gene. He has mild dysmorphism, hypotonia, developmental delay but not hypertrophic cardiomyopathy and tubulopathy which differ from other majority of reported patients. Therefore, hypertrophic cardiomyopathy and tubulopathy may not be considered as constant features of MRPS22. With this case report, we also present first symmetrical bilateral brainstem and medial thalamic lesions, and cerebellar and cerebral atrophy on a brain MR imaging follow-up of ten months.

摘要

MRPS22 基因缺陷是一种非常罕见的新发现的线粒体疾病。我们报告了一例 4 个月大的严重受累男性婴儿,其存在 MRPS22 突变。外显子组测序显示 MRPS22 基因的一个新的纯合剪接突变 c.339+5G>A。他有轻度的畸形、张力减退、发育迟缓,但没有肥厚型心肌病和肾小管病,这与其他大多数报道的患者不同。因此,肥厚型心肌病和肾小管病可能不被认为是 MRPS22 的恒定特征。通过本病例报告,我们还在 10 个月的脑磁共振成像随访中首次发现双侧脑桥和内侧丘脑对称病变,以及小脑和大脑萎缩。

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