BiochemistrybioRxiv
Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies
V. Venkatesan, A. C. Christopher, P. Babu…·S. Thangavel
This study edits the PRR-βE1 region in HSPCs to suppress HbA and enhance HbF production, showing therapeutic benefits for β-hemoglobinopathies.
DOI 10.1016/j.omtn.2023.04.024