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Systemic administration of 2-hydroxypropyl-β-cyclodextrin to symptomatic Npc1-deficient mice slows cholesterol sequestration in the major organs and improves liver function
In Niemann-Pick type C (NPC) disease, loss of function mutations in either NPC1 or NPC2 result in progressive accumulation of unesterified cholesterol (UC) and glycosphingolipids in all organs leading to neurodegeneration, pulmonary dysfunction and sometimes liver failure. There is no cure for this...
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| Publicado no: | Clin Exp Pharmacol Physiol |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2014
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4526211/ https://ncbi.nlm.nih.gov/pubmed/25115571 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/1440-1681.12285 |
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