In-depth characterization of stem cell potency and genotoxicity for clinical-scale ex vivo CRISPR-Cas9 gene editing

Molecular Therapy Advances 2026 Open access

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Authors: Asma Naseem, Winston Vetharoy, Thomas E. Whittaker, Federica Zinghirino, Antonio Carusillo, Roopkatha Ghosh, Eduardo Gomez Castaneda, Hashim Ali, Carlo Cipriani, Eugenio Montini, Adrian J. Thrasher, Giorgia Santilli
+3 more Daniela Cesana, Giandomenico Turchiano, Alessia Cavazza
DOI: 10.1016/j.omta.2026.201816
Cited by: 1
References: 42

Abstract

chromosomal rearrangement that could be detected exclusively following cell engraftment in mice. This work offers a robust and adaptable roadmap for future HDR-based gene editing platforms, establishing critical benchmarks for efficacy, safety, and regulatory readiness in the development of advanced therapeutic medicinal products.

Fields of Study & Subjects

CRISPR and Genetic Engineering Pluripotent Stem Cells Research Single-cell and spatial transcriptomics

Publication details

Journal / source
Molecular Therapy Advances
Publisher
Elsevier BV
Volume
34
Issue
3
Pages
201816–201816
Publication date
22 July 2026
Language
EN