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Author

Andrea Cosentino

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Open access Jul 2026

Non-genotoxic transplantation and in vivo selection through epitope editing

Epitope editing of KIT enables antibody-based, non-genotoxic conditioning that selectively enriches therapeutic BCL11A-edited haematopoietic stem/progenitor cells, supports durable engraftment, preserves clonal diversity and enhances induction of fetal haemoglobin, a therapeutic approach for conditions such as sickle cell disease and β-thalassemia.

G. Casirati, Andrea Cosentino, Marta Freschi et al. · 0 citations
Open access Aug 2026

Functional restoration of immune defects in STAT1 gain-of-function disease following stem cell gene editing.

Efficient and precise correction of STAT1 GOF mutations by base editing is demonstrated, with maintenance of the correction through long-term engraftment in vivo, which represents the first application of gene editing to correct a dominant gain-of-function mutation causing immunodeficiency.

Robert Torrance, K. Orf, Nathan White et al. · 0 citations

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