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Abbreviations AAV: Adeno-associated virus ABC: ATP-binding cassette BBB: Bloodbrain barrier CNS: Central nervous system CSF: Cerebrospinal fluid ERT: Enzyme replacement therapy g7: Simil-opioid glycopeptide GAG: Glycosaminoglycan IDDD: Intrathecal drug delivery device LSD: Lysosomal storage disorder mAb: Monoclonal antibody MPS: Mucopolysaccharidosi(e)s NP: Nanoparticle SLC: Solute carriers TfR: Transferrin receptor TJ: Tight junction References Lampe C, Bellettato CM, Karabul N, Scarpa M

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