EDSpliCE, a CRISPR-Cas9 gene editing platform to rescue splicing, effectively corrects inherited retinal dystrophy-associated splicing defects
Pietro De Angeli, Stefanida Shliaga, Arturo Flores-Tufiño, Eleonora Roschi, Salome Spaag, Katarína Štingl, Laura Kuehlewein, Bernd Wissinger, Susanne Kohl
EDSpliCE corrects splicing defects in inherited retinal dystrophy using CRISPR-Cas9 with a single gRNA and exonuclease, showing effective splicing rescue in minigene assays and patient-derived fibroblasts.
bioRxiv · Mar 27, 2024 · PreprintGenomics