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[DOI] [PMC free article] [PubMed] [Google Scholar] 86.Mettler S.K., Nath H.P., Grumley S., Orejas J.L., Dolliver W.R., Nardelli P., Yen A.C., Kligerman S.J., Jacobs K., Manapragada P.P., et al

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Researchers developing brain-targeting therapies for Sanfilippo have a few options: they can administer a therapy directly into the CNS, adapt a therapy to cross the BBB from the blood, or use a delivery vehicle to carry a therapy across the BBB