Disrupting ZBTB7A or BCL11A binding sites reactivates fetal hemoglobin in erythroblasts from healthy and β0-thalassemia/HbE individuals

Disrupting ZBTB7A or BCL11A binding sites reactivates fetal hemoglobin in erythroblasts from healthy and β0-thalassemia/HbE individuals

Abstract CRISPR/Cas9 genome editing has emerged as a promising treatment for genetic diseases like β-thalassemia. Editing γ-globin promoters to disrupt ZBTB7A/LRF or BCL11A binding sites has shown potential for reactivating fetal hemoglobin and treating sickle cell disease. However, its application...

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Main Authors: Chokdee Wongborisuth, Pawarit Innachai, Chonticha Saisawang, Alisa Tubsuwan, Natee Jearawiriyapaisarn, Pavita Kaewprommal, Jittima Piriyapongsa, Wararat Chiangjong, Usanarat Anurathapan, Duantida Songdej, Amornrat Tangprasittipap, Suradej Hongeng
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Scientific Reports
Subjects:
CRISPR/Cas9 genome editing
γ-globin promoter
Fetal hemoglobin reactivation
β0-thalassemia/HbE
ZBTB7A/LRF
BCL11A
Online Access:https://doi.org/10.1038/s41598-025-10791-8
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https://doi.org/10.1038/s41598-025-10791-8

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