Simon N. Chu

This paper develops a combinatorial base editing strategy to enhance erythroid lineage amplification and disease correction in hematopoietic stem and progenitor cells.

This paper develops small molecule-inducible EPO receptors to enable erythropoiesis without recombinant EPO, reducing costs and improving scalability of RBC manufacturing.

This paper describes a genome editing strategy that restores hemoglobin production in α-thalassemia major-derived hematopoietic stem and progenitor cells by integrating a functional α-globin gene and a truncated erythropoietin receptor.