Base editing HbS to HbG-Makassar improves hemoglobin function supporting its use in sickle cell disease.

doi:10.1038/s41467-025-56578-3

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Article

Base editing HbS to HbG-Makassar improves hemoglobin function supporting its use in sickle cell disease.

Nature communications - 7 Feb 2025

Kostamo Zachary, Ortega Manuel A, Xu Chavonna, Feliciano Patricia R, Budak Elizabeth, Lam Daisy, Winton Valerie, Jenkins Rebecca, Venugopal Archita, Zhang Margaret, Jamieson John, Coisman Brent, Goldsborough Kennedy, Hernandez Britney, Kanne Celeste K, Evans Erica N, Zgodny Jordan, Zhang Yankai, Darazim Jawa, Patel Ashwin, Pendergast Michael A, Manis John, Hartigan Adam J, Ciaramella Giuseppe, Lee Seung-Joo, Chu S Haihua, Sheehan Vivien A

Abstract excerpt

Adenine base editing can convert sickle hemoglobin (HbS, βΕ6V) to G-Makassar hemoglobin (HbG, βE6A), a naturally occurring variant that is clinically asymptomatic. However, the quality and functionality of purified HbG and of mature HbGG and HbGS red blood cells (RBC) has not been assessed. Here, we develop a mouse model to characterize HbG. Purified HbG appears normal and does not polymerize under hypoxia. The...

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PMID: 39920120
DOI: 10.1038/s41467-025-56578-3
PMCID: PMC11806015

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