GTF2H5基因突变的3型毛硫营养不良:阿根廷1例报告

IF 0.7 4区 医学 Q4 PEDIATRICS
Jimena Dri, Eugenia Dos Santos, Adriana Fernández, Florencia Galdeano, María J Guillamondegui, Cristina Gatica
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引用次数: 0

摘要

毛硫营养不良症是一种罕见的神经外胚层缺陷,其特征是毛发稀疏、脆性、光敏、智力残疾和身材矮小。在西方国家,发病率为百万分之1.2,报告的病例中有一半具有与一般转录因子IIH复合物的三个亚基突变相关的临床和细胞光敏性,这涉及转录和核苷酸切除修复。已报道6例GTF2H5突变患者;这是在阿根廷的第一份报道。患者3岁时偏光显微镜“虎尾带”诊断,9岁时分子生物学证实。她表现出生长迟缓,发育迟缓和体重不足比报道的更为严重。鉴于患病率低,临床异质性高,早期诊断、跨学科管理和遗传咨询需要高度的怀疑指数。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Trichothiodystrophy type 3 with a mutation in the GTF2H5 gene: A case report in Argentina.

Trichothiodystrophy is a rare neuroectodermal defect characterized by sparse and brittle hair, photosensitivity, intellectual disability, and short stature. With an incidence of 1.2 per million in Western countries, half of the reported cases have clinical and cellular photosensitivity associated with mutations in three subunits of the general transcription factor IIH complex, which is involved in transcription and nucleotide excision repair. Six patients with GTF2H5 mutations have been reported; this is the first report in Argentina. The patient was diagnosed at 3 years of age by "tiger tail banding" on polarized light microscopy, and at 9 years of age, it was confirmed by molecular biology. She presented growth retardation with more severe stunting and underweight than reported. Given the low prevalence and high clinical heterogeneity, a high index of suspicion is required for early diagnosis, interdisciplinary management, and genetic counseling.

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来源期刊
CiteScore
1.40
自引率
25.00%
发文量
286
审稿时长
6-12 weeks
期刊介绍: Archivos Argentinos de Pediatría is the official publication of the Sociedad Argentina de Pediatría (SAP) and has been published without interruption since 1930. Its publication is bimonthly. Archivos Argentinos de Pediatría publishes articles related to perinatal, child and adolescent health and other relevant disciplines for the medical profession.
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