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, M. K. K. Azhagiri, K. Walavalkar, B. Saravanan, S. Srinivasan, K. V. Karuppusamy, A. Jacob, S. Rangaraj, A. Bagchi, A. A. Pai, Y. Nakamura, P. Balasubramanian, R. Pai, , S. Thangavel
This study edits the PRR-βE1 region in HSPCs to suppress HbA and enhance HbF production, showing therapeutic benefits for β-hemoglobinopathies.
bioRxiv · Jul 11, 2022Biochemistry