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

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

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wetenschappelijk artikel

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наукова стаття, опублікована в червні 2018

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Controversies on the potential ...... al cholesterol storage disease

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Controversies on the potential ...... al cholesterol storage disease

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Controversies on the potential ...... al cholesterol storage disease

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Controversies on the potential ...... al cholesterol storage disease

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Controversies on the potential ...... al cholesterol storage disease

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Controversies on the potential ...... al cholesterol storage disease

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P1476

Controversies on the potential ...... al cholesterol storage disease

@en

P2093

Juan F Calderón

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P2860

Inhibition of mTOR induces autophagy and reduces toxicity of polyglutamine expansions in fly and mouse models of Huntington disease

Rapamycin selectively inhibits interleukin-2 activation of p70 S6 kinase

Anatomically defined neuron-based rescue of neurodegenerative Niemann-Pick type C disorder.

Impaired proteolysis underlies autophagic dysfunction in Niemann-Pick type C disease

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.

Disruption and therapeutic rescue of autophagy in a human neuronal model of Niemann Pick type C1

Modeling diseases in multiple mouse strains for precision medicine studies.

Npc1 deficiency in the C57BL/6J genetic background enhances Niemann-Pick disease type C spleen pathology.

P304

135-136

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P31

scholarly article

P356

10.1016/J.YMGMR.2018.05.001

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P407

English

P478

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P50

ANDRES KLEIN

P577

2018-06-01T00:00:00Z

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P698

30023307

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P921

endocrinology

molecular biology

P932

6047216

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