Durable Correction of Phenylketonuria In Vivo Following a Single Intravenous Dose of AAVHSC15 Packaging a Human Phenylalanine Hydroxylase Transgene
Ahmed S, Ellsworth JL, Francone O, et al. ESGCT 2018.
Ahmed S, Ellsworth JL, Francone O, et al. ESGCT 2018.