Laura Breda

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 identifies translation inhibitors that enable ex vivo expansion of human hematopoietic stem cells while preserving their function and reducing cytokine exposure.

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.