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Xmn1 -158 γG变异体与重型β地中海贫血和镰状细胞贫血的严重程度及胎儿血红蛋白水平的关联

Association of Xmn1 -158 γG variant with severity and HbF levels in β-thalassemia major and sickle cell anaemia.

作者信息

Dadheech Sneha, Jain Suman, Madhulatha D, Sharma Vandana, Joseph James, Jyothy A, Munshi Anjana

机构信息

Centre for Human Genetics, Central University of Punjab, Bathinda, Punjab, 151001, India.

出版信息

Mol Biol Rep. 2014 May;41(5):3331-7. doi: 10.1007/s11033-014-3195-5. Epub 2014 Feb 2.

Abstract

Haemoglobinopathies including β-thalassemia and sickle cell anaemia (SCA) are considered to be classical monogenic diseases. There is considerable clinical variability between patients inheriting identical β-globin mutations. The reasons for this variability are not well understood. Previous studies have suggested that a variety of genetic determents influence different clinical phenotypes. The genetic variants that modulate HbF levels have a very strong impact on ameliorating the clinical phenotype. In the present study 6,500 blood samples from suspected cases were analysed using HPLC, ARMS-PCR, RDB techniques. Patients with β-thalassemia and SCA were classified into mild, moderate, severe according to the severity score based on Hb levels, age of onset, age at which patients received their first blood transfusion, the degree of growth retardation and splenectomy. Patients with β-thalassemia and SCA were analysed for Xmn1 polymorphism and association between this polymorphism and severity of β-thalassemia and SCA was evaluated. We found a significant difference in genotypic and allelic frequencies of Xmn1 polymorphism between mild and moderate and mild and severe cases. There was a significant difference in high and low percentage of HbF in CC, CT and TT bearing individuals. The TT bearing individuals were found to have a high percentage of HbF in β-thalassemia as well as SCA. This study confirms that increased γG-globin expression associated with Xmn1 polymorphism ameliorates the clinical severity in β-thalassemia as well as SCA in the study population.

摘要

血红蛋白病,包括β地中海贫血和镰状细胞贫血(SCA),被认为是典型的单基因疾病。携带相同β珠蛋白突变的患者之间存在相当大的临床变异性。这种变异性的原因尚不清楚。先前的研究表明,多种遗传因素影响不同的临床表型。调节HbF水平的基因变异对改善临床表型有非常强烈的影响。在本研究中,使用高效液相色谱法(HPLC)、扩增阻滞突变系统聚合酶链反应(ARMS-PCR)、反向点杂交技术(RDB)对6500份疑似病例的血样进行了分析。根据基于血红蛋白水平、发病年龄、首次输血年龄、生长发育迟缓程度和脾切除术的严重程度评分,将β地中海贫血和SCA患者分为轻度、中度、重度。对β地中海贫血和SCA患者进行Xmn1多态性分析,并评估该多态性与β地中海贫血和SCA严重程度之间的关联。我们发现轻度与中度、轻度与重度病例之间Xmn1多态性的基因型和等位基因频率存在显著差异。携带CC、CT和TT的个体中HbF高低百分比存在显著差异。发现携带TT的个体在β地中海贫血和SCA中均有较高百分比的HbF。本研究证实,与Xmn1多态性相关的γG-珠蛋白表达增加可改善研究人群中β地中海贫血和SCA的临床严重程度。

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