In-Depth Characterization of Stem Cell Potency and Genotoxicity for Clinical-Scale Ex Vivo CRISPR/Cas9 Gene Editing
Asma Naseem, Winston Vetharoy, Thomas E. Whittaker, Federica Zinghirino, Antonio Carusillo, Roopkatha Ghosh, Eduardo Gómez‐Castañeda, Hashim Ali, Carlo Cipriani, Eugenio Montini, Adrian James Thrasher, Giorgia Santilli, Daniela Cesana, Giandomenico Turchiano, Alessia Cavazza
This study develops a clinical-compliant framework for ex vivo CRISPR/Cas9 gene editing of HSPCs to correct WAS, showing improved engraftment and a favorable safety profile with minimal off-target effects.
Molecular Therapy Advances · Jul 22, 2026Peer-reviewedMolecular Biology