SPLICER: A Highly Efficient Base Editing Toolbox That Enables In Vivo Therapeutic Exon Skipping
Angelo Miskalis, Shraddha Shirguppe, Jackson Winter, Gianna Elias, Devyani Swami, Ananthan Nambiar, Michelle Stilger, Wendy S. Woods, Nicholas C. Gosstola, Michael Gapinske, M. Alejandra Zeballos C., Hayden J. Moore, Sergei Maslov, Thomas Gaj, Pablo Pérez‐Piñera
SPLICER improves exon skipping by editing both splice acceptor and donor sites, reducing aberrant splicing and enabling therapeutic applications in Alzheimer's disease.
Nature Communications · Nov 28, 2024Peer-reviewedBioengineering