Author

I. Merelli

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#gene editing Open access Sep 2026

p38 MAPK inhibition safeguards genome integrity during gene editing of hematopoietic stem and progenitor cells.

Ex vivo genome editing of hematopoietic stem and progenitor cells (HSPCs) holds significant therapeutic potential but remains constrained by genotoxic risks associated with nuclease-induced DNA double-strand breaks, DNA donor template delivery and sensing, and proliferation-induced stress during ex vivo manipulation. T...

Roberta Vacca, Lucrezia della Volpe, Nicolò Gualandi et al. · Molecular Therapy · 0 citations

Aug 2026

Locus-specific transposable element expression drives human hematopoietic stem cell disease pathophysiology

This study provides direct evidence that VEXAS-specific TE govern HSC clonal dominance, thereby uncovering a regulatory axis underlying HSC biology and disease mechanisms, opening a therapeutic strategy directed towards the repetitive genome.

A. Varesi, Sontago Dong, Chiara Gaddoni et al. · bioRxiv · 0 citations