鉴定 HBB 基因中的一个新变体 c.163delG,该变体导致一名中型地中海贫血患者出现 Beta 缺失表型。

IF 1.2 4区 医学 Q4 BIOCHEMISTRY & MOLECULAR BIOLOGY Hemoglobin Pub Date : 2024-01-01 Epub Date: 2024-01-23 DOI:10.1080/03630269.2023.2279609
M Mamata, G Padma, T Pragna Laxmi, K Saroja, Dalal Ashwin, Jain Suman
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引用次数: 0

摘要

一名 21 岁的患者既往病史为面色苍白、轻度黄疸、乏力加重、血红蛋白低、血红蛋白变异分析异常,输血超过 70 次。他被转诊进行基因分析,以确定β-球蛋白基因的致病变异。对该患者及其家族进行的桑格测序发现,在血红蛋白 E(HbE)(HBB:c.79G > A)变异的复合杂合状态下,存在一个新的框架转换变异 HBB:c.163delG。患者的父亲和兄弟姐妹的 HBB 基因正常。母亲是 HbE(HBB:c.79G > A)变异体的杂合子。通过 Mutalyzer 进行的硅分析预测,c.163delG 变异在七个密码子之后产生了一个过早的终止密码子,导致蛋白质截短。FoldX 蛋白质稳定性分析表明,ΔΔG 值为 45.27 千卡/摩尔,表明蛋白质稳定性下降。HBB:c.79G > A 是一种已知的 HbE 变异编码变异,会导致β-球蛋白链合成减少,表现为轻度地中海贫血。该患者体内的 HBB:c.163delG 和 HBB:c.79G > A 变体共同作用,可能会导致β-球蛋白链合成减少,从而出现轻型地中海贫血的临床表现。
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Identification of a Novel Variant c.163delG in HBB Gene Resulting in a Beta Null Phenotype in a Proband with Thalassemia Intermedia.

A 21-year-old patient presented with a previous medical history of pallor, mild icterus, increased fatigue, low hemoglobin, and abnormal hemoglobin variant analysis with more than 70 transfusions. He was referred for genetic analysis to identify the pathogenic variations in the β-globin gene. Sanger's sequencing of the proband and his family revealed the presence of a novel frame shift variant HBB:c.163delG in a compound heterozygous state with hemoglobin E (HbE) (HBB:c.79G > A) variant. The father and the sibling of the patient were found to be normal for the HBB gene. Mother was found to be heterozygous for HbE (HBB:c.79G > A) variant. In silico analysis by Mutalyzer predicted that c.163delG variant generated a premature stop codon after seven codons, leading to a truncated protein. FoldX protein stability analysis showed a positive ΔΔG value of 45.27 kcal/mol suggesting a decrease in protein stability. HBB:c.79G > A is a known variant coding for HbE variant, which results in the reduced synthesis of β-globin chain and shows mild thalassemia. Combined effect of HBB:c.163delG and HBB:c.79G > A variants in the proband might have led to the reduced synthesis of β-globin chains resulting in a thalassemia intermedia type of clinical manifestation.

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来源期刊
Hemoglobin
Hemoglobin 医学-生化与分子生物学
CiteScore
1.70
自引率
10.00%
发文量
59
审稿时长
3 months
期刊介绍: Hemoglobin is a journal in the English language for the communication of research and information concerning hemoglobin in humans and other species. Hemoglobin publishes articles, reviews, points of view The journal covers topics such as: structure, function, genetics and evolution of hemoglobins biochemical and biophysical properties of hemoglobin molecules characterization of hemoglobin disorders (variants and thalassemias), consequences and treatment of hemoglobin disorders epidemiology and prevention of hemoglobin disorders (neo-natal and adult screening) modulating factors methodology used for diagnosis of hemoglobin disorders
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