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Viola Carzaniga

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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