Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease
Varun Katta, Kiera O’Keefe, Yichao Li, Thiyagaraj Mayuranathan, Cícera Regina Lazzarotto, Rachael K. Wood, Rachel M. Levine, Alicia Powers, Kalin D. Mayberry, Garret Manquen, Yu Yao, Jingjing Zhang, Yoonjeong Jang, Nikitha Nimmagadda, Erin Dempsey, , Shengdar Q. Tsai
This study develops a genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease, showing efficient editing, therapeutic HbF levels, and no off-target effects.
Molecular Therapy · Jul 30, 2024Peer-reviewedGenomics