Skip to main content
. Author manuscript; available in PMC: 2015 Apr 30.
Published in final edited form as: Sci Transl Med. 2015 Feb 25;7(276):276ra26. doi: 10.1126/scitranslmed.3010101

Fig 2. Liver histology and biochemistry in NPC cats administered SC or IC HPβCD.

Fig 2

(A) Untreated NPC cats showed severe vacuolization of hepatocyte and Kupffer cell cytoplasm that was ameliorated by 1000 mg/kg HPβCD but was unaffected by 120 mg IC HPβCD. All cats were 24 weeks of age; scale bar = 100 microns. (B) HPTLC of total lipids in 2 mg tissue samples. Migration in chloroform-methanol-H2O 65:25:4, anisaldehyde spray. The prominent storage of cholesterol and sphingomyelin was greatly reduced in all SC-treated cats but remained unchanged with IC administration. (C) HPTLC showing a similar effect of treatment on neutral glycosphingolipids from 5 mg tissue samples after saponification of total lipids. Migration as in B; stained with orcinol-H2SO4 spray. (D) Free sphingosine concentrations (measured by HPLC) that were increased ~50-fold in untreated NPC cats, decreased ~5 times after SC-HPβCD treatment. For the SC graph, both cats received 8000 mg/kg HPβCD. For the IC graph, data from cats receiving 15 mg, 30 mg, and 60 mg were included as there was no dose effect. Normal values: 37±15 pmol/mg protein (mean ± SD, n=7). Abbreviations: Untr’d: untreated; Norm: normal; SC8, SC4, SC1: subcutaneous, 8000, 4000 or 1000 mg/kg; IC120, IC60: intracisternal, 120 or 60 mg; Chol: unesterified cholesterol; BMP: bis(monoacylglycero)phosphate; Sph: sphingomyelin; GlcCer: glucosylceramide; Lac Cer: lactosylceramide; Gb3: globotriaosylceramide; HPTLC, high performance thin-layer chromatography; HPLC, high pressure liquid chromatography.