Ontology highlight
ABSTRACT:
SUBMITTER: Kao ML
PROVIDER: S-EPMC7317750 | biostudies-literature | 2020 May
REPOSITORIES: biostudies-literature
Kao Mark L ML Stellar Susan S Solon Eric E Lordi Alfred A Kasica Nicole N Swain Gary G Bagel Jessica H JH Gurda Brittney L BL Vite Charles H CH
Journal of inherited metabolic disease 20191215 3
2-Hydroxypropyl-β-cyclodextrin (HP-β-CD) is an experimental therapy for Niemann-Pick disease type C (NPC) that reduced neuronal cholesterol and ganglioside storage, reduced Purkinje cell death, and increased lifespan in npc1-/- mice and NPC1 cats. In this study, tissue distribution was investigated in normal cats that received a single 120-mg dose of [<sup>14</sup> C]-HP-β-CD (approximately 200 μCi/cat) via the cerebellomedullary cistern (CBMC) and lumbar cistern. One cat was euthanized at each ...[more]