A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.
2016
Ronzitti, G., et al. (2016).
Mol Ther Methods Clin Dev 3: 16049.
2016
Ronzitti, G., et al. (2016).
Mol Ther Methods Clin Dev 3: 16049.