Credit: Peter Bell, PhD, Perelman School of Medicine, University of Pennsylvania
This image shows efficient restoration of OTC expression in the liver of mice treated at neonatal stage by AAV8.CRISPR-SaCas9-mediated gene correction. Immunofluorescence staining with antibodies against OTC on liver sections from an 8-week-old mouse treated with the dual AAV vectors for CRISPR-SaCas9-mediated gene correction as a newborn.