AAVHSC15 Packaging Human Phenylalanine Hydroxylase Results in Sustained In Vivo Correction of Phenylketonuria Following a Single IV Administration in the Murine Model

AAVHSC15 Packaging Human Phenylalanine Hydroxylase Results in Sustained In Vivo Correction of Phenylketonuria Following a Single IV Administration in the Murine Model

Ahmed SS, Wang M, Ellsworth JL, et al.