Today we’re highlighting one of our saporin conjugates, Anti-CD117-SAP. This is a tool for depleting cells that express CD117, the tyrosine kinase growth factor receptor.
CD117 is expressed on hematopoietic stem cells, mast cells, and acute myeloid leukemia cells. Some of the areas where CD117 plays an essential role is in the regulation of cell survival and proliferation, hematopoiesis, stem cell maintenance and mast cell development, migration and function.
Allogeneic hematopoietic stem cell transplantation can cure genetic diseases such as severe combined immunodeficiency (SCID), but it’s associated with significant toxicities such as graft-versus-host disease (GvHD). Using a patients’ own gene-modified hematopoietic stem and progenitor cells (HSPCs) can eliminate the risk of GvHD, but the treatment relies on gene transfer using viruses and genotoxic conditioning which carries risks.
The authors describe how base-editing is an alternative means to correct genetic defects while engineered virus-like particles (eVLPs) can deliver the base-edited proteins without the risks seen with viral integration.
In conclusion, transplantation of HSPCs that have been repaired through eVLP-mediated base-editing with CD117-ADC conditioning successfully reversed the SCID phenotype in mice, and highlights a significant advancement in disease treatment without the current toxicities.
