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, Jessica Zuin, Alessandro Sinopoli, Anna Camus, Thomas Burgold, Ewelina Magdalena Bartoszek, Diego Calabrese, Mylene Toranelli, Julia Rositzka, Manuel Rhiel, Geoffroy Andrieux, Chang Long Li, Alvin Hsu, , Lukas T. Jeker
This paper demonstrates a method using CD117-blocking antibody and epitope-engineered HSPCs to treat β-thalassemia without myeloablative conditioning.
bioRxiv · Jul 8, 2026 · PreprintBioengineering