“…Among these are different dosages and routes of administration of ERT [103,125,222,229,[231][232][233][234][235], microcapsules enclosing myoblasts over-expressing IDS [183], different gene-therapy approaches [164][165][166]171,172,223,236], genistein [188], brain-penetrating IgG-Iduronate 2-sulphatase fusion protein [134], HSCT [234,237,238], ZFN-mediated in vivo genome editing [239], engineered nanoparticles for IDS enzyme brain-targeting [184,185], anti-human transferrin receptor antibody fusion protein [135], and chloroquine [230]. They were also useful for testing new methods of GAG analysis, aimed at improving their use as biomarkers in pre-clinical studies and successively in patients [125,223,240,241]. In particular, the RP-HPLC method [242] was applied to analyse the total relative amount of HS and its disaccharide composition [223], while another HPLC-based approach, founded on an analysis of the 2-sulfoiduronic acid derived from the non-reducing end of GAGs, allowed clear discrimination between Ids-ko and wild-type mice in the liver and brain [240].…”