Cargando…
2-Hydroxypropyl-gamma-cyclodextrin overcomes NPC1 deficiency by enhancing lysosome-ER association and autophagy
Niemann-Pick type C (NPC) disease is a fatal neurodegenerative disorder caused by mutations in NPC1 and NPC2 genes that result in an accumulation of cholesterol in lysosomes. The majority of children with NPC die in adolescence. Currently, no FDA-approved therapies exist for NPC and the mechanisms o...
Autores principales: | Singhal, Ashutosh, Krystofiak, Evan S., Jerome, W. Gray, Song, Byeongwoon |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2020
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7250861/ https://www.ncbi.nlm.nih.gov/pubmed/32457374 http://dx.doi.org/10.1038/s41598-020-65627-4 |
Ejemplares similares
-
Hydroxypropyl-beta and -gamma cyclodextrins rescue cholesterol accumulation in Niemann–Pick C1 mutant cell via lysosome-associated membrane protein 1
por: Singhal, Ashutosh, et al.
Publicado: (2018) -
Differential Effects of 2-Hydroxypropyl-Cyclodextrins on Lipid Accumulation in Npc1-Null Cells
por: Hoque, Sanzana, et al.
Publicado: (2020) -
Cyclodextrins: Assessing the Impact of Cavity Size, Occupancy, and Substitutions on Cytotoxicity and Cholesterol Homeostasis
por: Szente, Lajos, et al.
Publicado: (2018) -
A marked paucity of granule cells in the developing cerebellum of the Npc1(−/−) mouse is corrected by a single injection of hydroxypropyl-β-cyclodextrin
por: Nusca, S., et al.
Publicado: (2014) -
Influence of Npc1 genotype on the toxicity of hydroxypropyl-β-cyclodextrin, a potentially therapeutic agent, in Niemann–Pick Type C disease models()
por: Tanaka, Yuta, et al.
Publicado: (2014)