Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2018 May 12:15:135-136.
doi: 10.1016/j.ymgmr.2018.05.001. eCollection 2018 Jun.

Controversies on the potential therapeutic use of rapamycin for treating a lysosomal cholesterol storage disease

Affiliations

Controversies on the potential therapeutic use of rapamycin for treating a lysosomal cholesterol storage disease

Juan F Calderón et al. Mol Genet Metab Rep. .
No abstract available

PubMed Disclaimer

Figures

Fig. 1
Fig. 1
Rapamycin exerts opposite effects on NPC mice depending on their genetic background. Kaplan-Meier survival curves (in days) of Npc1−/− mice from the C57 (A) and FVB (B) genetic backgrounds daily i.p. injected with Mock (solid lines) or Rapamycin (Rapa) (10 mg/kg) (dashed lines). Significant differences in survival curves were determined by Log-rank (Mantel–Cox) analyses. Values were considered statistically significantly different when p ≤ 0.05. Animal studies are in compliance with regulations set by our Institutional Animal Care and Use Committee.

References

    1. Elrick M.J., Yu T., Chung C., Lieberman A.P. Impaired proteolysis underlies autophagic dysfunction in Niemann-Pick type C disease. Hum. Mol. Genet. 2012;21:4876–4887. - PMC - PubMed
    1. Paulina Ordonez M., Roberts E.A., Kidwell C.U., Yuan S.H., Plaisted W.C., Goldstein L.S.B. Disruption and therapeutic rescue of autophagy in a human neuronal model of Niemann pick type C1. Hum. Mol. Genet. 2012;21:2651–2662. - PMC - PubMed
    1. Maetzel D., Sarkar S., Wang H., Abi-Mosleh L., Xu P., Cheng A.W., Gao Q., Mitalipova M., Jaenisch R. Genetic and chemical correction of cholesterol accumulation and impaired autophagy in hepatic and neural cells derived from niemann-pick type C patient-specific iPS cells. Stem Cell Rep. 2014;2:866–880. - PMC - PubMed
    1. Parra J., Klein A.D., Castro J., Morales M.G., Mosqueira M., Valencia I., Cortés V., Rigotti A., Zanlungo S. Npc1 deficiency in the C57BL/6J genetic background enhances Niemann-Pick disease type C spleen pathology. Biochem. Biophys. Res. Commun. 2011;413:400–406. - PubMed
    1. Lopez M.E., Klein A.D., Dimbil U.J., Scott M.P. Anatomically defined neuron-based rescue of neurodegenerative Niemann-Pick type C disorder. J. Neurosci. 2011;31:4367–4378. - PMC - PubMed

LinkOut - more resources