In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene
Marco De Giorgi, So Hyun Park, Adam B. Castoreno, Mingming Cao, Ayrea E. Hurley, Lavanya Saxena, Marcel A. Chuecos, Christopher J. Walkey, Alexandria M. Doerfler, Mia N. Furgurson, M. Cecilia Ljungberg, Kalyani R. Patel, Sarah Hyde, Tyler Chickering, Stephanie Lefebvre, , William R. Lagor
This paper introduces a method to increase the proportion of correctly repaired hepatocytes in the liver through transient gene inhibition and transgene delivery, achieving a 25% increase in targeted cells and five-fold transgene expression.
Science Translational Medicine · Feb 12, 2025Peer-reviewedGenetics