SPLICER: A Highly Efficient Base Editing Toolbox That Enables In Vivo Therapeutic Exon Skipping
A. Miskalis, S. Shirguppe, J. Winter, G. Elias, D. Swami, A. Nambiar, M. Stilger, W. Woods, N. Gosstola, M. Gapinske, A. Zeballos, H. Moore, S. Maslov, T. Gaj, P. Perez-Pinera
SPLICER improves exon skipping by editing both splice acceptor and donor sites, reducing aberrant splicing and enabling therapeutic applications in Alzheimer's disease.
bioRxiv · Apr 1, 2024 · PreprintBioengineering