Gene regulation in erythropoiesis and therapeutic genome editing for genetic anemias
My research combines genome editing and red blood cell precursor biology to develop new therapies for genetic anemias, ranging from rare disorders such as α-thalassemia and congenital dyserythropoietic anemias to more prevalent conditions including sickle cell disease and β-thalassemia. I investigate advanced editing strategies, including prime editing, to design universal, mutation-agnostic genome editing approaches. In parallel, I study the transcriptional regulation of erythroid differentiation and ineffective erythropoiesis using technologies such as single-cell RNA sequencing and CRISPR-Cas9 screening, with the goal of identifying therapeutic targets that can be modulated genetically or pharmacologically.