Supplementary Materials

Supplementary Material for:

Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

Charles H. Vite,* Jessica H. Bagel, Gary P. Swain, Maria Prociuk, Tracey U. Sikora, Veronika M. Stein, Patricia O'Donnell, Therese Ruane, Sarah Ward, Alexandra Crooks, Su Li, Elizabeth Mauldin, Susan Stellar, Marc De Meulder, Mark L. Kao, Daniel S. Ory, Cristin Davidson, Marie T. Vanier, Steven U. Walkley

*Corresponding author. E-mail: vite@vet.upenn.edu

Published 25 February 2015, Sci. Transl. Med. 7, 276ra26 (2015)
DOI: 10.1126/scitranslmed.3010101

This PDF file includes:

  • Fig. S1. Weekly mean body weights of normal control, untreated NPC, and NPC cats administered SC or IC HPβCD.
  • Fig. S2. Liver gangliosides and brain glucosylceramide.
  • Fig. S3. Immunofluorescence staining of GM2 ganglioside in marginal gyrus of cerebral cortex.
  • Fig. S4. Immunofluorescence staining of MBP in cerebellar white matter.
  • Fig. S5. Abnormal posture and MRI of shoulders of 76-week-old NPC cat that received both SC and IC HPβCD.
  • Legends for videos S1 to S5

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Other Supplementary Material for this manuscript includes the following:

  • Video S1 (.mov format). Untreated 21-week-old NPC cat showing tremor (+3), ataxia (+4), and inability to stand without support.
  • Video S2 (.mov format). NPC cat (22 weeks old) administered HPβCD (8000 mg/kg SC) weekly since 3 weeks of age showing mild head tremor (+1) and ataxia (+1).
  • Video S3 (.mov format). NPC cat (24 weeks old) administered 120 mg of HPβCD IC biweekly since 3 weeks of age appearing neurologically normal.
  • Video S4 (.mov format). NPC cat (76 weeks old) administered 120 mg of HPβCD IC biweekly since 3 weeks of age showing no head tremor and mild ataxia (+1).
  • Video S5 (.mov format). NPC cat (24 weeks old) administered 120 mg of HPβCD IC biweekly since 16 weeks of age showing head tremor (+2) and ataxia (+2).

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