BioengineeringMolecular TherapyPeer-reviewed
T cell correction pipeline for Inborn Errors of Immunity
Katariina Mamia, Sólrún Kolbeinsdóttir, Kornel Labun…·Emma Maria Haapaniemi
This paper presents a T cell gene correction pipeline using CRISPR/Cas9 and HDR to correct monogenic mutations in IEI models, achieving up to 80% bi-allelic editing with no detectable off-target effects.