Article

Installation of HbG-Makassar by base editing restores hemoglobin function: a transformative therapy for sickle cell disease

2024-03-14

Abstract excerpt

<title>Abstract</title> Adenine base editing offers a viable gene-based therapy for sickle cell disease (SCD), converting sickle hemoglobin (HbS, βΕ6V) to G-Makassar hemoglobin (HbG, βE6A), a naturally occurring, non-pathogenic variant. However, HbG functionality alone and with HbS has been largely uncharacterized. We present a mouse model used to characterize purified HbG-Makassar as well as HbGG and HbGS red...

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Literature Corpus work: ba475f8b-782e-5efc-b324-48d21ad1b9ad
DOI: 10.21203/rs.3.rs-3995314/v1

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