Abstract

Sickle cell disease (SCD) is an inherited blood disorder marked by homozygosity of hemoglobin S, which is a defective hemoglobin caused by a missense mutation in the β-globin gene. However, clinical phenotypes of SCD vary among patients. To investigate genetic variants associated with various clinical phenotypes of SCD, we genotyped DNA samples from 520 SCD subjects and used a genome-wide association study (GWAS) approach to identify genetic variants associated with phenotypic features of SCD. For HbF levels, the previously reported 2p16.1 locus (BCL11A) reached genome significance (rs1427407, P = 8.58 × 10–10) in our GWAS as expected. In addition, we found a new genome-wide significance locus at 15q14 (rs8182015, P = 2.07 × 10–8) near gene EMC7. GWAS of acute chest syndrome (ACS) detected a locus (rs79915189, P = 3.70 × 10–8) near gene IDH2 at 15q26.1. The SNP, rs79915189, is also an expression quantitative trait locus (eQTL) of IDH2 in multiple tissues. For vasoocclusive episode (VOE), GWAS detected multiple significant signals at 2p25.1 (rs62118798, P = 4.27 × 10–8), 15q26.1 (rs62020555, P = 2.04 × 10–9) and 15q26.3 (rs117797325, P = 4.63 × 10–8). Our findings provide novel insights into the genetic mechanisms of SCD suggesting that common genetic variants play an important role in the presentation of the clinical phenotypes of patients with SCD.

Details

Title
Identification of genetic variants associated with clinical features of sickle cell disease
Author
Tsukahara, Katharine 1 ; Chang, Xiao 2 ; Mentch, Frank 2 ; Smith-Whitley, Kim 3 ; Bhandari, Anita 3 ; Norris, Cindy 3 ; Glessner, Joseph T. 4 ; Hakonarson, Hakon 4 

 Children’s Hospital of Philadelphia, Division of Pulmonary and Sleep Medicine, Philadelphia, USA (GRID:grid.239552.a) (ISNI:0000 0001 0680 8770) 
 Research Institute, Leonard Madlyn Abramson Research Center, The Center for Applied Genomics, Children’s Hospital of Philadelphia, Philadelphia, USA (GRID:grid.239552.a) (ISNI:0000 0001 0680 8770) 
 Children’s Hospital of Philadelphia, Division of Hematology, Philadelphia, USA (GRID:grid.239552.a) (ISNI:0000 0001 0680 8770); Children’s Hospital of Philadelphia, Division of Pulmonary and Sleep Medicine, Philadelphia, USA (GRID:grid.239552.a) (ISNI:0000 0001 0680 8770) 
 Research Institute, Leonard Madlyn Abramson Research Center, The Center for Applied Genomics, Children’s Hospital of Philadelphia, Philadelphia, USA (GRID:grid.239552.a) (ISNI:0000 0001 0680 8770); University of Pennsylvania School of Medicine, Department of Pediatrics, Philadelphia, USA (GRID:grid.25879.31) (ISNI:0000 0004 1936 8972) 
Pages
20070
Publication year
2024
Publication date
2024
Publisher
Nature Publishing Group
e-ISSN
20452322
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
3098519044
Copyright
© The Author(s) 2024. This work is published under http://creativecommons.org/licenses/by-nc-nd/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.