GenomicsMolecular TherapyPeer-reviewed
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…·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.
18 citations