Gene therapy in advanced metachromatic leukodystrophy: tempering expectations

Schoenmakers, Daphne H.; Beerepoot, Shanice; Adang, Laura A.; Asbreuk, Marije A.B.C.; Bergner, Caroline G.; Bley, Annette E.; Boelens, Jaap Jan; Calbi, Valeria; Darling, Alejandra; Eklund, Erik; García Cazorla, Ángeles; Grønborg, Sabine W.; Groeschel, Samuel; van Hasselt, Peter M.; Hollak, Carla E.M.; Horgan, Claire; Jones, Simon; de Koning, Tom; Laugwitz, Lucia; Lindemans, Caroline; Martin, Pascal; Mochel, Fanny; Øberg, Andreas; Ram, Dipak; Sevin, Caroline; Schöls, Ludger; Zerem, Ayelet; Wolf, Nicole I.; Fumagalli, Francesca