Nuclease-Free Genome Editing by AAVHSC Vectors Leads to In Vivo Genome Correction and Amelioration of Disease Phenotype in a Mouse Model of Phenylketonuria (PKU)

Nuclease-Free Genome Editing by AAVHSC Vectors Leads to In Vivo Genome Correction and Amelioration of Disease Phenotype in a Mouse Model of Phenylketonuria (PKU)

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