AAVHSC Nuclease-Free Genome Editing Leads to In Vivo Genome Correction and a Significant Reduction in Disease Phenotype in a Mouse Model of Phenylketonuria

AAVHSC Nuclease-Free Genome Editing Leads to In Vivo Genome Correction and a Significant Reduction in Disease Phenotype in a Mouse Model of Phenylketonuria

Wright J, Smith L, Adamson-Small L, et al.