Nuclease-Free and Promoter-Less AAVHSC-Mediated Genome Editing In Vivo Corrects the Disease Phenotype in a Mouse Model of Phenylketonuria
Wright J, Ellsworth JL, St. Martin T, et al. ASGCT 2019.
Wright J, Ellsworth JL, St. Martin T, et al. ASGCT 2019.