Dib C, Queenan JA, Willner H, Swartzrock L, Charlesworth CT, Denis M, Davis JR, Mepani RJ, Ho K, Castro M, Wilson RC, Nakauchi H, Liu DR, Czechowicz A (2026) Restoration of the immune system with base editing and non-genotoxic conditioning in a Rag2 point-mutant mouse model. Mol Ther 34(7):4070-4083. doi: 10.1016/j.ymthe.2026.04.010 PMID: 41968583
Objective: To investigate therapy that would eliminate the risks associated with current hematopoietic stem cell (HSC) gene-modification and conditioning approaches.
Summary: The authors combined base editors, engineered virus-like particles, and non-genotoxic CD117-SAP conditioning to explore optimal curative treatment of severe combined immunodeficiency. Results showcased a significant advancement in reducing treatment-related toxicities while enabling disease correction.
Usage: Using a mouse model, Anti-CD117 was combined with Streptavidin-ZAP (IT-27) and administered at 1.5 mg/kg via intravenous injection 8 days before transplantation.
Related Products: Streptavidin-ZAP (Cat. #IT-27), Anti-CD117-SAP (Cat. #IT-83)