Department of Medicine, Boston University School of Medicine, Boston, MA, USA.
Blood. 2011 May 5;117(18):4935-45. doi: 10.1182/blood-2010-11-317081. Epub 2011 Mar 8.
Fetal hemoglobin (HbF) is regulated as a multigenic trait. By genome-wide association study, we confirmed that HBS1L-MYB intergenic polymorphisms (HMIP) and BCL11A polymorphisms are highly associated with HbF in Chinese β-thalassemia heterozygotes. In this population, the variance in HbF resulting from the HMIP is 13.5%; that resulting from the BCL11A polymorphism is 6.4%. To identify the functional variant in HMIP, we used 1000 Genomes Project data, single nucleotide polymorphism imputation, comparisons of association results across populations, potential transcription factor binding sites, and analysis of phylogenetic conservation. Based on these studies, a hitherto unreported association between HbF expression and a 3-bp deletion, between 135 460 326 and 135 460 328 bp on chromosome 6q23 was found. This 3-bp deletion is in complete linkage disequilibrium with rs9399137, which is the single nucleotide polymorphism in HMIP most significantly associated with HbF among Chinese, Europeans, and Africans. Chromatin immunoprecipitation assays confirmed erythropoiesis-related transcription factors binding to this region in K562 cells. Based on transient expression of a luciferase reporter plasmid, the DNA fragment encompassing the 3-bp deletion polymorphism has enhancer-like activity that is further augmented by the introduction of the 3-bp deletion. This 3-bp deletion polymorphism is probably the most significant functional motif accounting for HMIP modulation of HbF in all 3 populations.
胎儿血红蛋白 (HbF) 作为一个多基因性状受到调控。通过全基因组关联研究,我们证实 HBS1L-MYB 基因间多态性 (HMIP) 和 BCL11A 多态性与中国 β-地中海贫血杂合子中的 HbF 高度相关。在该人群中,HMIP 导致的 HbF 变异为 13.5%;BCL11A 多态性导致的 HbF 变异为 6.4%。为了确定 HMIP 中的功能变体,我们使用了 1000 基因组计划数据、单核苷酸多态性推断、跨人群关联结果的比较、潜在转录因子结合位点以及系统发生保守性分析。基于这些研究,我们发现了一个以前未报道的与 HbF 表达相关的 3-bp 缺失,该缺失位于 6q23 染色体上的 135460326 和 135460328bp 之间。该 3-bp 缺失与 rs9399137 完全连锁不平衡,后者是在中国、欧洲和非洲人群中与 HbF 关联最显著的 HMIP 中的单核苷酸多态性。染色质免疫沉淀实验证实了该区域与 K562 细胞中与红细胞生成相关的转录因子的结合。基于瞬时表达荧光素酶报告质粒,包含 3-bp 缺失多态性的 DNA 片段具有增强子样活性,进一步通过引入 3-bp 缺失而增强。该 3-bp 缺失多态性可能是最显著的功能基序,可解释 HMIP 在所有 3 个人群中对 HbF 的调控。