Giuliana Ferrari

This study uses a genetic anemia model to show that reduced thrombopoietin affects hematopoietic stem cells and the bone marrow niche through direct and indirect mechanisms.

Preclinical safety and efficacy studies of a new gene therapy approach for patients with severe β-hemoglobinopathies.

This study develops a bifunctional lentiviral vector for sickle cell disease that combines gene addition and gene silencing to reduce HbS levels and improve treatment efficacy.