LNP-mediated BCL11A Editing Corrects Sickling Phenotypes and Preserves HSPC Fitness Compared to Electroporation
Yaw Ofosu Nyansa Ansong‐Ansongton, Corynne Stéphanie Ahouéfa Adanho, Utku Goreke, Atip Lawanprasert, Mikhail Vysotskiy, York Tang, Alissa LW Kleinhez, Wilson S. Ross, Angela E. Rivers, Niren Murthy, David N. Nguyen
This study compares LNP and electroporation for delivering CRISPR to HSPCs, finding LNPs provide better cell viability and HbF induction with lower toxicity.
bioRxiv · Aug 31, 2026 · PreprintBioengineering