Stefano Rivella

This paper develops a novel lentiviral vector for gene therapy of MLD that achieves high ARSA activity with minimal genomic integrations, improving safety and efficacy in preclinical models.

This study shows that SUMF1 gene therapy via hematopoietic stem cell transplant improves biochemical and functional outcomes in a mouse model of Multiple Sulfatase Deficiency.

This paper investigates the role of p21 in β-thalassemia erythroid apoptosis and shows that while reducing p21 or Foxo3 decreases apoptosis, it does not improve ineffective erythropoiesis.