Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrin
D. Carradori, H. Chen, B. Werner, A. Shah, C. Leonardi, M. Usuelli, R. Mezzenga, F. M. Platt, J.-C. Leroux
This study investigates the mechanism of crosslinked 2-hydroxypropyl-β-cyclodextrin (HPβCD) in treating Niemann-Pick Disease Type C and evaluates its in vivo efficacy with MRIg-FUS to enhance brain penetration.
bioRxiv · Jul 31, 2020 · PreprintPharmacology & Toxicology