Nuclease-Free and Promoter-Less AAVHSC-Mediated Genome Editing In Vivo Corrects the Disease Phenotype in a Mouse Model of Phenylketonuria

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.