Kiriaki Paschoudi

This study demonstrates in vivo gene therapy for hemoglobinopathies using EPOR editing to enhance the expansion of gene-corrected erythroid cells without drug selection.

This paper demonstrates a method using CD117-blocking antibody and epitope-engineered HSPCs to treat β-thalassemia without myeloablative conditioning.

The paper identifies potent, compact, and lineage-specific enhancers for gene therapy vectors using epigenomic data and validates their efficacy in correcting a thalassemia phenotype.