Morgado Ana, Picanço Isabel, Gomes Susana, Miranda Armandina, Coucelo Margarida, Seuanes Filomena, Seixas Maria Teresa, Romão Luísa, Faustino Paula
Centro de Genética Humana, Instituto Nacional de Saúde Dr Ricardo Jorge, Lisboa, Portugal.
Eur J Haematol. 2007 Nov;79(5):422-8. doi: 10.1111/j.1600-0609.2007.00949.x. Epub 2007 Oct 4.
The phenotype of increased Hb A2 typical of beta-thalassaemia (beta-thal) carriers can be reduced to normal or borderline values because of the co-inheritance of a delta-globin gene (HBD, MIM #142000) mutation, which may lead to misinterpretation of diagnostic results. To know the spectrum of delta-globin mutations in the Portuguese population we performed a mutational analysis of the delta-globin gene in a group of 51 Portuguese beta-thal carriers presenting microcytosis, hypochromia and a normal/borderline Hb A2 level and in another group of 15 individuals suspected to have delta-globin structural abnormalities. The heterozygosity for the beta(+)IVS-I-6T-->C (HBB:c. 92+6T>C) mutation was the main cause for the mentioned atypical beta-thal carrier phenotype. Furthermore, eight individuals were double heterozygous for one common beta-thal mutation and the delta(+)Cd27G-->T mutation (Hb A2-Yialousa; HBD:c.82G>T). One of them also presented a novel delta-globin gene promoter mutation,-80G-->A (HBD:c.-130G>A), responsible for about 25% decrease of the promoter activity in transient expression assays. One the other hand, in the other group of 15 individuals suspected to have delta-globin structural abnormalities observed by biochemical methods, some known Hb A2 variants were identified - Hb A2' (HBD:c.49G>C), Hb A2-Babinga (HBD:c.410G>A), and Hb A2-Wrens (HBD:c.295G>A), and the novel Hb A2-Fogo [delta64(E8)(Gly-->Ser); (HBD:c.193G>A)]. This novel Hb A2 variant was observed segregating in linkage with Hb E (HBB:c.79G>A) in a three generation family. In conclusion, six different delta-globin mutations were found, being two of them new molecular defects. All delta-alleles identified were found linked to the expected beta-globin cluster haplotype. All mutations caused a low Hb A2 level and through this could lead to misdiagnosis when inherited together with a beta-thal allele.
由于共同遗传了δ-珠蛋白基因(HBD,MIM #142000)突变,β地中海贫血(β-地贫)携带者典型的Hb A2升高表型可降至正常或临界值,这可能导致诊断结果的错误解读。为了解葡萄牙人群中δ-珠蛋白突变谱,我们对一组51名表现为小红细胞症、低色素血症和正常/临界Hb A2水平的葡萄牙β-地贫携带者以及另一组15名疑似有δ-珠蛋白结构异常的个体进行了δ-珠蛋白基因的突变分析。β(+)IVS-I-6T→C(HBB:c. 92+6T>C)突变的杂合性是上述非典型β-地贫携带者表型的主要原因。此外,8名个体为一种常见β-地贫突变和δ(+)Cd27G→T突变(Hb A2-Yialousa;HBD:c.82G>T)的双重杂合子。其中一人还出现了一种新的δ-珠蛋白基因启动子突变,-80G→A(HBD:c.-130G>A),在瞬时表达试验中该突变导致启动子活性降低约25%。另一方面,在通过生化方法观察到疑似有δ-珠蛋白结构异常的另一组15名个体中,鉴定出了一些已知的Hb A2变异体——Hb A2'(HBD:c.49G>C)、Hb A2-Babinga(HBD:c.410G>A)和Hb A2-Wrens(HBD:c.295G>A),以及新的Hb A2-Fogo [δ64(E8)(Gly→Ser);(HBD:c.193G>A)]。在一个三代家族中观察到这种新的Hb A2变异体与Hb E(HBB:c.79G>A)连锁分离。总之,发现了六种不同的δ-珠蛋白突变,其中两种是新的分子缺陷。所有鉴定出的δ-等位基因均与预期的β-珠蛋白基因簇单倍型连锁。所有突变均导致Hb A2水平降低,因此当与β-地贫等位基因一起遗传时可能导致误诊。