Open access
LNP-mediated BCL11A Editing Corrects Sickling Phenotypes and Preserves HSPC Fitness Compared to Electroporation
Yaw Ansong-Ansongton
C. S. A. Adanho
Utku Goreke
A. Lawanprasert
M. Vysotskiy
York Tang
Alissa LW Kleinhez
Ross C. Wilson
Angela Rivers
N. Murthy
David N. Nguyen
Biology
TL;DR
Although RNP electroporation achieves higher CRISPR editing rates, it reduces HSPC recovery and RBC differentiation; conversely, mRNA-LNP delivery achieves modest rates while preserving HSPC fitness, progenitor expansion, and RBC enucleation efficiencies.