Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease-Reference-Cited by-同舟云学术

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

Published:2015-02-25 Issue:276 Volume:7 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Vite Charles H.¹,Bagel Jessica H.¹,Swain Gary P.¹,Prociuk Maria¹,Sikora Tracey U.²,Stein Veronika M.¹,O’Donnell Patricia²,Ruane Therese²,Ward Sarah¹,Crooks Alexandra¹,Li Su¹,Mauldin Elizabeth²,Stellar Susan³,De Meulder Marc⁴,Kao Mark L.³,Ory Daniel S.⁵,Davidson Cristin⁶,Vanier Marie T.⁷,Walkley Steven U.⁶

Affiliation:

1. Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.

2. Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.

3. Janssen Research & Development, LLC, Janssen Pharmaceutical Companies of Johnson and Johnson, Titusville, NJ 08560, USA.

4. Janssen Research & Development, a division of Janssen Pharmaceutica NV, Janssen Pharmaceutical Companies of Johnson and Johnson, Beerse, Belgium.

5. Diabetic Cardiovascular Disease Center, Washington University School of Medicine, St. Louis, MO 63110, USA.

6. Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Intellectual and Developmental Disabilities Research Center, Albert Einstein College of Medicine, Bronx, NY 10461, USA.

7. INSERM U820; EA4611, Université Claude Bernard Lyon 1, Lyon, France.

Abstract

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.

Funder

NIH

Ara Parseghian Medical Research Foundation

National Niemann-Pick Disease Foundation

German Research Foundation

Dana's Angels Research Trust

Race for Adam

Niemann-Pick Type C Disease

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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