In this application note, we demonstrate the performance of Pin-point cytidine base editors (CBEs) in HSPCs in comparison to traditional CRISPR-Cas9 editing. We show that base editing with the Pin-point platform achieves efficient and specific editing, while better preserving cell viability and the most primitive, repopulating HSC fraction — critical for the development of durable, autologous cell therapies.
The Pin-point™ base editing platform technology is available for clinical or diagnostic study and commercialization under a commercial license from Revvity.
Safe and efficient base editing in hematopoietic stem and progenitor cells: Avoiding the pitfalls of DNA breaks