Lama Raju, Yusof Wardah, Shrestha Tilak R, Hanafi Sarifah, Bhattarai Matrika, Hassan Rosline, Zilfalil Bin Alwi
Central Department of Biotechnology, Tribhuvan University, Kirtipur, Kathmandu, Nepal.
Malaysian Node of Human Variome Project, School of Medical Sciences, Universiti Sains Malaysia, Kelantan, Malaysia.
Hematol Oncol Stem Cell Ther. 2022 Mar 1;15(1):279-284. doi: 10.1016/j.hemonc.2021.01.004.
Beta-thalassemia is a genetic disorder that is inherited in an autosomal recessive pattern. This genetic disease leads to a defective beta-globin hemoglobin chain causing partial or complete beta-globin chain synthesis loss. Beta-thalassemia major patients need a continuous blood transfusion and iron chelation to maintain the normal homeostasis of red blood cells (RBCs) and other systems in the body. Patients also require treatment procedures that are costly and tedious, resulting in a serious health burden for developing nations such as Nepal.
A total of 61 individuals clinically diagnosed to have thalassemia were genotyped with multiplex amplification refractory mutation system-polymerase chain reaction (ARMS-PCR). Twenty-one major mutations were investigated using allele-specific primers grouped into six different panels.
The most common mutations found (23%) were IVS 1-5 (G-C) and Cd 26 (G-A) (HbE), followed by 619 deletion, Cd 8/9 (+G), Cd 16 (-C), Cd 41/42 (-TTCT), IVS 1-1 (G-T), Cd 19 (A-G), and Cd 17 (A-T) at 20%, 12%, 8%, 6%, 4%, 3%, and 1%, respectively.
The results of this study revealed that Nepal's mutational profile is comparable to that of its neighboring countries, such as India and Myanmar. This study also showed that thalassemia could be detected across 17 Nepal's ethnic groups, especially those whose ancestors originated from India and Central Asia.
β地中海贫血是一种以常染色体隐性模式遗传的基因紊乱疾病。这种基因疾病会导致β珠蛋白血红蛋白链缺陷,造成部分或全部β珠蛋白链合成缺失。重型β地中海贫血患者需要持续输血和铁螯合治疗,以维持体内红细胞(RBC)和其他系统的正常稳态。患者还需要昂贵且繁琐的治疗程序,这给尼泊尔等发展中国家带来了沉重的健康负担。
对61名临床诊断为地中海贫血的个体进行多重扩增阻滞突变系统 - 聚合酶链反应(ARMS-PCR)基因分型。使用分为六个不同组别的等位基因特异性引物研究了21种主要突变。
发现最常见的突变(23%)是IVS 1-5(G-C)和Cd 26(G-A)(HbE),其次是619缺失、Cd 8/9(+G)、Cd 16(-C)、Cd 41/42(-TTCT)、IVS 1-1(G-T)、Cd 19(A-G)和Cd 17(A-T),分别占20%、12%、8%、6%、4%、3%和1%。
本研究结果表明,尼泊尔的突变谱与其邻国如印度和缅甸的突变谱相当。该研究还表明,在尼泊尔的17个民族中都能检测到地中海贫血,尤其是那些祖先来自印度和中亚的民族。