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Author

A. Varesi

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#gene editing Oct 2026

Editing cells in place: Targeted in vivo gene editing of hematopoietic stem cells.

Traditional ex vivo hematopoietic stem cell (HSC) gene therapy faces manufacturing challenges. A recent study1 in Nature Biomedical Engineering demonstrates that antibody-engineered lipid nanoparticles achieve efficient and durable in vivo editing of HSCs in humanized mice, offering a promising therapeutic strategy for...

Sijin Luozhong, A. Varesi, Bo-Wen Li · 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. · 0 citations

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