Single Intravenous Dose of AAVHSC15 Packaging a Human Phenylalanine Hydroxylase Transgene Results in Durable Correction of Phenylketonuria In Vivo

Single Intravenous Dose of AAVHSC15 Packaging a Human Phenylalanine Hydroxylase Transgene Results in Durable Correction of Phenylketonuria In Vivo

Francone OL, Ahmed SS, Ellsworth JL, et al.