[1例1型糖尿病患者纤维结石性胰腺糖尿病与SPINK1基因突变相关的临床和遗传特征]。

W X Zhao, H Wang, B Huang, S W Li, K L Feng, J Q Cui, M Liu
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引用次数: 0

摘要

为探讨2024年6月在天津医科大学总医院机场就诊的1型糖尿病(T1DM)合并纤维结石性胰腺糖尿病患者的发病机制,对其进行临床和遗传特征分析。通过全外显子组测序(WES)筛选潜在致病基因,Sanger测序验证了家族内鉴定的遗传变异。先证者血糖水平升高,酪氨酸磷酸酶抗体阳性,提示诊断为T1DM。先证者胰腺可见多发钙化。基因检测显示先证者携带两个丝氨酸肽酶抑制剂Kazal 1型(SPINK1)基因变异,分别为C .194+2T>C和C . 215g >A。根据美国医学遗传学与基因组学学会(ACMG)指南,将C .194+2T>C突变初步归类为致病性,而C . 215g >A突变被归类为不确定意义变异(VUS)。生物信息学分析表明,SPINK1基因的C .194+2T>C变异导致了一个截断的蛋白,影响了该蛋白的三维结构和活性。该突变与c - 215g >A突变完全连锁不平衡,可能具有保护功能,影响临床表型。考虑到患者同时患有T1DM和FCPD,应提高对两种糖尿病合并症的认识,以防止误诊和漏诊。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
[Clinical and genetic characteristics of fibrocalculous pancreatic diabetes associated with SPINK1 gene mutations in a patient with type 1 diabetes].

To investigate the pathogenic mechanisms of a patient with type 1 diabetes mellitus (T1DM) complicated with fibrocalculous pancreatic diabetes at Tianjin Medical University General Hospital Airport Site in June 2024, clinical and genetic characteristic analyses were performed. Potential pathogenic genes were screened by whole-exome sequencing (WES), and Sanger sequencing validated the identified genetic variants within the family. The proband exhibited elevated blood glucose levels and positivity for tyrosine phosphatase antibodies, suggesting a diagnosis of T1DM. Multiple calcifications in the pancreas were observed in the proband. Genetic testing revealed that the proband carried two variants in the serine peptidase inhibitor Kazal type 1 (SPINK1) gene, namely, c.194+2T>C and c.-215G>A. According to the American College of Medical Genetics and Genomics (ACMG) guidelines, the c.194+2T>C mutation is preliminarily classified as pathogenic, while the c.-215G>A variant is classified as a variant of uncertain significance (VUS). Bioinformatics analysis indicated that the c.194+2T>C variant in the SPINK1 gene results in a truncated protein, affecting the three-dimensional structure and activity of the protein. This mutation is in complete linkage disequilibrium with the c.-215G>A variant, which may have a protective function and influence the clinical phenotype. Given that the patient also has T1DM and FCPD, there should be increased awareness of the co-occurrence of both types of diabetes to prevent misdiagnosis and underdiagnosis.

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