Genetic and Functional Insights into F-Cell Distribution in Sickle Cell Anemia: A Genome-Wide Association Study Approach

镰状细胞贫血中F细胞分布的遗传和功能见解:全基因组关联研究方法

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Abstract

BACKGROUND: Sickle cell anemia (SCA) is a severe hereditary disorder characterized by hemoglobin polymerization, vaso-occlusion, and chronic hemolysis. The distribution of F-cells, which retain fetal hemoglobin (HbF), plays a crucial role in mitigating disease severity. Genome-wide association studies (GWAS) have identified genetic determinants influencing F-cell persistence, yet the underlying mechanisms require further elucidation. OBJECTIVE: This study aims to analyse publicly available GWAS data to identify key genetic loci associated with F-cell distribution in SCA, assess their biological relevance, and explore potential therapeutic implications. METHODS: GWAS data were extracted from publicly available repositories and analysed for significant single nucleotide polymorphisms (SNPs) associated with F-cell levels. Functional enrichment analysis was performed using Gene Ontology (GO) and Kyoto Encyclopedia of Genes and Genomes (KEGG) pathway analysis. Transcription factor (TF) interactions, microRNA (miRNA) regulation, and protein-protein interactions (PPI) were explored to understand the molecular mechanisms governing HbF expression. RESULTS: The study identified BCL11A, HBS1L-MYB, and β-globin cluster polymorphisms as primary regulators of F-cell distribution. Functional analysis highlighted pathways related to erythropoiesis, chromatin remodelling, and transcriptional regulation. miRNA-target interactions and epigenetic modifiers emerged as critical determinants of HbF persistence. CONCLUSION: These findings provide insights into the genetic regulation of F-cell distribution, reinforcing the therapeutic potential of HbF modulation. Future studies should focus on functional validation and targeted interventions to enhance HbF expression in SCA. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s12288-025-02127-3.

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