Open access
Jul 2026
In-depth characterization of stem cell potency and genotoxicity for clinical-scale ex vivo CRISPR-Cas9 gene editing
A clinical-compatible platform enabling large-scale manufacturing while preserving HSPC viability, stemness, and multilineage functionality is established and a robust and adaptable roadmap for future HDR-based gene editing platforms is offered.
A. Naseem, W. Vetharoy, T. E. Whittaker et al.
· Molecular therapy. Advances · 1 citation