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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
Aug 2026 · bioRxiv · 0 citations · 6 references
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.

DOI Semantic Scholar
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