Fais Antonella, Casu Mariano, Ruggerone Paolo, Ceccarelli Matteo, Porcu Simona, Era Benedetta, Anedda Roberto, Sollaino Maria Carla, Galanello Renzo, Corda Marcella
Department of Sciences Applied to Biosystems, University of Cagliari, S.P. Monserrato-Sestu km 0.700, 09042 Monserrato, Italy.
ISRN Hematol. 2011;2011:735314. doi: 10.5402/2011/735314. Epub 2010 Nov 29.
WE REPORT THE FIRST CASE OF COSEGREGATION OF TWO HAEMOGLOBINS (HBS): HbG-Philadelphia [α68(E17)Asn → Lys] and HbDuarte [β62(E6)Ala → Pro]. The proband is a young patient heterozygous also for β°-thalassaemia. We detected exclusively two haemoglobin variants: HbDuarte and HbG-Philadelphia/Duarte. Functional study of the new double variant HbG-Philadelphia/Duarte exhibited an increase in oxygen affinity, with a slight decrease of cooperativity and Bohr effect. This functional behaviour is attributed to β62Ala → Pro instead of α68Asn → Lys substitution. Indeed, HbG-Philadelphia isolated in our laboratory from blood cells donor carrier for this variant is not affected by any functional modification, whereas purified Hb Duarte showed functional properties very similar to the double variant. NMR and MD simulation studies confirmed that the presence of Pro instead of Ala at the β62 position produces displacement of the E helix and modifications of the tertiary structure. The substitution α68(E17)Asn → Lys does not cause significant structural and dynamical modifications of the protein. A possible structure-based rational of substitution effects is suggested.
我们报告了首例两种血红蛋白(HbS)共分离的病例:HbG-费城[α68(E17)Asn→Lys]和Hb杜阿尔特[β62(E6)Ala→Pro]。先证者是一名年轻患者,也是β°地中海贫血的杂合子。我们仅检测到两种血红蛋白变体:Hb杜阿尔特和HbG-费城/杜阿尔特。新的双变体HbG-费城/杜阿尔特的功能研究显示氧亲和力增加,协同性和玻尔效应略有降低。这种功能行为归因于β62Ala→Pro取代,而非α68Asn→Lys取代。实际上,我们实验室从该变体的血细胞供体携带者中分离出的HbG-费城不受任何功能修饰的影响,而纯化的Hb杜阿尔特显示出与双变体非常相似的功能特性。核磁共振(NMR)和分子动力学(MD)模拟研究证实,β62位的Pro取代Ala会导致E螺旋移位和三级结构改变。α68(E17)Asn→Lys取代不会引起蛋白质显著的结构和动力学修饰。本文提出了基于结构的取代效应的可能原理。