Molecular BiologyMolecular Therapy AdvancesPeer-reviewed
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…·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.
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