Division of Neonatology, Case Western Reserve University, Cleveland, USA.
N Engl J Med. 2011 May 12;364(19):1837-43. doi: 10.1056/NEJMoa1013579.
Globin-gene mutations are a rare but important cause of cyanosis. We identified a missense mutation in the fetal Gγ-globin gene (HBG2) in a father and daughter with transient neonatal cyanosis and anemia. This new mutation modifies the ligand-binding pocket of fetal hemoglobin by means of two mechanisms. First, the relatively large side chain of methionine decreases both the affinity of oxygen for binding to the mutant hemoglobin subunit and the rate at which it does so. Second, the mutant methionine is converted to aspartic acid post-translationally, probably through oxidative mechanisms. The presence of this polar amino acid in the heme pocket is predicted to enhance hemoglobin denaturation, causing anemia.
珠蛋白基因突变是导致发绀的一个罕见但重要的原因。我们在一名患有短暂性新生儿发绀和贫血的父女中发现了胎儿 Gγ-珠蛋白基因(HBG2)的错义突变。这种新的突变通过两种机制改变了胎儿血红蛋白的配体结合口袋。首先,甲硫氨酸较大的侧链降低了氧与突变血红蛋白亚基结合的亲和力和结合速度。其次,突变的甲硫氨酸可能通过氧化机制在翻译后被转化为天冬氨酸。预测这种极性氨基酸在血红素口袋中的存在会增强血红蛋白变性,导致贫血。