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)

Ellsworth J, Smith L, St. Martin T, et al.