GALC and Krabbe disease: The severity of the disease and extent of gene replacement required to treat Krabbe disease is underscored by the fact that even in combination with bone marrow transplant it required neonatal IV, IT and intraparenchymal AAV9-CAGGS-GALC to achieve extension of lifespan to 9 months with long-term improvement of neurological and physiological signs (Marshall et al., 2018).