Mayuranathan Thiyagaraj, Rayabaram Janakiram, Das Reena, Arora Neeraj, Edison Eunice S, Chandy Mammen, Srivastava Alok, Velayudhan Shaji R
Department of Haematology, Christian Medical College, Vellore, India.
Eur J Haematol. 2014 Jun;92(6):514-20. doi: 10.1111/ejh.12276. Epub 2014 Mar 15.
Hereditary persistence of foetal haemoglobin (HPFH) and (δβ)(0) -thalassaemia are conditions caused by large deletions that involve δ- and β-globin genes in the β-globin cluster, and they are characterized by increased haemoglobin (HbF) levels in adults. Significant phenotypic diversity is observed between the different mutations that cause these conditions. Molecular characterization of these deletions is important for accurate molecular diagnosis, and they will also provide the information on the cis-acting genetic regulatory elements present in the β-globin cluster.
We performed gap-PCR, multiplex ligation-dependent probe amplification (MLPA), quantitative fluorescent multiplex PCR (QF-MPCR) and DNA sequencing to detect and characterize the deletions in the β-globin cluster.
We characterized six different deletions resulting in (δβ)(0) -thalassaemia or HPFH in 51 unrelated families.
With the help of multiple genetic tools, we performed comprehensive genetic analysis of HPFH and (δβ)(0) -thalassaemia in Indian population and could define the molecular basis of these conditions in this population. We also identified two novel HPFH mutations, 49.98 kb (HPFH-9) and 86.7 kb (HPFH-10) deletions, in this population.
胎儿血红蛋白遗传性持续存在(HPFH)和(δβ)(0)-地中海贫血是由涉及β-珠蛋白基因簇中δ-和β-珠蛋白基因的大片段缺失引起的疾病,其特征是成人血红蛋白(HbF)水平升高。在导致这些疾病的不同突变之间观察到显著的表型多样性。这些缺失的分子特征对于准确的分子诊断很重要,并且它们还将提供有关β-珠蛋白基因簇中存在的顺式作用遗传调控元件的信息。
我们进行了缺口PCR、多重连接依赖探针扩增(MLPA)、定量荧光多重PCR(QF-MPCR)和DNA测序,以检测和表征β-珠蛋白基因簇中的缺失。
我们在51个无关家庭中鉴定出六种不同的缺失,导致(δβ)(0)-地中海贫血或HPFH。
借助多种遗传工具,我们对印度人群中的HPFH和(δβ)(0)-地中海贫血进行了全面的遗传分析,并确定了该人群中这些疾病的分子基础。我们还在该人群中鉴定出两种新的HPFH突变,即49.98 kb(HPFH-9)和86.7 kb(HPFH-10)缺失。