BioengineeringbioRxivPreprint
CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates β-thalassemia model
Romina Marone, Rosalba Lepore, Kiriaki Paschoudi…·Lukas T. Jeker
This paper demonstrates a method using CD117-blocking antibody and epitope-engineered HSPCs to treat β-thalassemia without myeloablative conditioning.