The team of researchers guided by Giuliana Ferrari, Group Leader of the Gene Transfer into Stem Cell lab at the San Raffaele-Telethon Institute for Gene Therapy and Full Professor in Molecular Biology at Vita-Salute San Raffaele University, discovered that altered TGF-β signaling leads to reduced dormancy and activation of blood stem and progenitor cells, which become skewed towards erythroid differentiation, which remains ineffective because of the genetic defect, in patients with β-thalassemia.
