Single Intravenous Administration of AAVHSC15 Packaging a Human Phenylalanine Hydroxylase Transgene Sustainably Corrects Phenylketonuria in Mouse Model

Single Intravenous Administration of AAVHSC15 Packaging a Human Phenylalanine Hydroxylase Transgene Sustainably Corrects Phenylketonuria in Mouse Model

Ahmed S, Benard L, Wang M, et al.