Precision and efficacy of RNA-guided DNA integration in high-expressing muscle loci
M. H. Padmaswari, G. N. Bulliard, S. Agrawal, M. S. Jia, C. E. Nelson
This study evaluates CRISPR-based gene integration in skeletal muscle using two high-expressing loci, demonstrating high-level therapeutic gene expression in mouse myoblasts.
bioRxiv · Mar 18, 2024Bioengineering