Intravenous delivery of AAVPHP.eB-hARSA-HA in MLD mice leads to a rapid and complete correction of brain sulfatide storage, one of the hallmarks of the disease (Gieselmann and Krägeloh-Mann, 2006), as our previous approach with intracerebral delivery of AAVrh10-ARSA (Piguet et al., 2012). This evidence concerns the gene ARSA and metachromatic leukodystrophy.