Protein Replacement Therapy Partially Corrects the Cholesterol-Storage Phenotype in a Mouse Model of Niemann-Pick Type C2 Disease

Niemann-Pick type C2 (NPC2) disease is a fatal autosomal recessive neurovisceral degenerative disorder characterized by late endosomal-lysosomal sequestration of low-density lipoprotein derived cholesterol. The breach in intracellular cholesterol homeostasis is caused by deficiency of functional NPC...

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Main Authors: Nielsen, Gitte Krogh, Dagnaes-Hansen, Frederik, Holm, Ida Elisabeth, Meaney, Steve, Symula, Derek, Andersen, Niels Trolle, Heegaard, Christian Würtz
格式: Artigo
語言:Inglês
出版: Public Library of Science 2011
主題:
Research Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3207855/
https://ncbi.nlm.nih.gov/pubmed/22073306
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0027287
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