Azusa Matsubara

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.