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Author

G. Santilli

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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. · 1 citation
Review Open access Jul 2026

In vivo delivery strategies for therapeutic CRISPR genome editing

A comprehensive review of delivery modalities of CRISPR systems solely in vivo that underpin their therapeutic translation and outlines the remaining barriers to durable, tissue-selective, and broadly deployable CRISPR therapeutics is provided.

L. Martin, Jure Bohinc, Alessandra Recchia et al. · 0 citations