二氢嘧啶酶酶缺乏症和先天性孤立肾上腺皮质激素缺乏症:一名斯里兰卡男孩的双重基因诊断。

Shifaniya Banu Mohideen, Pitipanage Mihika Samindi Fernando, Christian Beetz, Sabine Schroder, Catarina Pereira, Senaka Gunatilleke, Pyara Rathnayake, Eresha Jasinge
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引用次数: 0

摘要

我们报告了一名因频繁呼吸暂停、喂养问题、肌张力低下以及2周大时磁共振成像显示左侧大脑中动脉梗塞而接受检查的男性患者。在对尿液进行有机酸分析后,初步诊断为二氢嘧啶酶(DPYS)缺乏症;二氢尿嘧啶、胸腺嘧啶和尿嘧啶的存在强烈提示了 DPYS 缺乏症。随后通过全外显子组测序进行基因评估,发现了两个不同的基因突变:DPYS 基因的同卵致病变体 c.1010T>C p.Leu337Pro,导致 DPYS 缺乏症;TBX19 基因的同卵致病变体 c.535C>T p.Arg179*,与常染色体隐性先天性孤立性肾上腺皮质激素缺乏症有关。目前,该患者 2 岁,患有大运动迟缓和癫痫发作障碍。我们认为,该患者的临床表型可能是两种基因突变混合表达的结果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Dihydropyrimidinase enzyme deficiency and congenital isolated adrenocorticotrophin deficiency: dual genetic diagnosis in a Sri Lankan boy.

We report on a male patient who was investigated for frequent apneic episodes, feeding problems, hypotonia, and left-sided middle cerebral artery infarction in the magnetic resonance imaging at 2 weeks of age. Primary diagnosis of dihydropyrimidinase (DPYS) deficiency was suspected following the analysis of urine for organic acid; DPYS deficiency was strongly suggested by the presence of dihydrouracil, thymine, and uracil. Subsequent genetic evaluation by whole exome sequencing revealed 2 separate mutations, homozygous pathogenic variant c.1010T>C p.Leu337Pro of the DPYS gene, resulting in DPYS deficiency, and homozygous pathogenic variant c.535C>T p.Arg179* of TBX19 gene, which is associated with autosomal recessive congenital isolated adrenocorticotrophic hormone deficiency. Currently, the patient is 2 years old, and he has gross motor retardation and seizure disorder. We suggest that the clinical phenotype of the proband can be a result of mixed expression of both mutations.

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