Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease

Gregory Heller, Michael S. Marshall, Yazan Issa, Jeffrey N. Marshall, Duc Nguyen, Emily Rue, Koralege C. Pathmasiri, Miriam S. Domowicz, Richard B. van Breemen, Leon M. Tai, Stephanie M. Cologna, Stephen J. Crocker, Maria I. Givogri, Mark S. Sands, Ernesto R. Bongarzone