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

• DOI: 10.1126/scitranslmed.3010101
• Publication Date: 2015-02-26T06:44:12Z
• Authors: 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
• Keywords: Inflammation; Aging; Calbindins; 0303 health sciences; Cell Death; Staining and Labeling; Injections, Subcutaneous; Liver Diseases; Fluorescent Antibody Technique; Alanine Transaminase; Auditory Threshold; G(M2) Ganglioside; Niemann-Pick Disease, Type C; General Medicine; 2-Hydroxypropyl-beta-cyclodextrin; 3. Good health; Purkinje Cells; 03 medical and health sciences; Liver; Cisterna Magna; Cats; Animals; Ataxia; Lung

Intracisternal injection of cyclodextrin into cats with Niemann-Pick type C1 disease results in Purkinje cell survival and normal neurological function, suggesting its usefulness for treating the human disease.