| Title |
Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression
|
|---|---|
| Published in |
PLOS ONE, September 2009
|
| DOI | 10.1371/journal.pone.0006951 |
| Pubmed ID | |
| Authors |
Cristin D. Davidson, Nafeeza F. Ali, Matthew C. Micsenyi, Gloria Stephney, Sophie Renault, Kostantin Dobrenis, Daniel S. Ory, Marie T. Vanier, Steven U. Walkley |
| Abstract |
Niemann-Pick type C (NPC) disease is a fatal neurodegenerative disorder caused most commonly by a defect in the NPC1 protein and characterized by widespread intracellular accumulation of unesterified cholesterol and glycosphingolipids (GSLs). While current treatment therapies are limited, a few drugs tested in Npc1(-/-) mice have shown partial benefit. During a combination treatment trial using two such compounds, N-butyldeoxynojirimycin (NB-DNJ) and allopregnanolone, we noted increased lifespan for Npc1(-/-) mice receiving only 2-hydroxypropyl-beta-cyclodextrin (CD), the vehicle for allopregnanolone. This finding suggested that administration of CD alone, but with greater frequency, might provide additional benefit. |
Mendeley readers
The data shown below were compiled from readership statistics for 206 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 1 | <1% |
| Portugal | 1 | <1% |
| Denmark | 1 | <1% |
| Germany | 1 | <1% |
| Unknown | 202 | 98% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Ph. D. Student | 42 | 20% |
| Researcher | 32 | 16% |
| Other | 23 | 11% |
| Student > Bachelor | 19 | 9% |
| Student > Master | 18 | 9% |
| Other | 29 | 14% |
| Unknown | 43 | 21% |
| Readers by discipline | Count | As % |
|---|---|---|
| Agricultural and Biological Sciences | 53 | 26% |
| Biochemistry, Genetics and Molecular Biology | 34 | 17% |
| Medicine and Dentistry | 26 | 13% |
| Pharmacology, Toxicology and Pharmaceutical Science | 12 | 6% |
| Neuroscience | 7 | 3% |
| Other | 22 | 11% |
| Unknown | 52 | 25% |