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Autophagy Volume 10, 2014 - Issue 6
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Autophagic Punctum

Restarting stalled autophagy a potential therapeutic approach for the lipid storage disorder, Niemann-Pick type C1 disease

Sovan Sarkar Whitehead Institute for Biomedical Research; Massachusetts Institute of Technology; Cambridge, MA USACorrespondence[email protected] [email protected]
,
Dorothea Maetzel Whitehead Institute for Biomedical Research; Massachusetts Institute of Technology; Cambridge, MA USA
,
Viktor I Korolchuk Institute for Ageing and Health; Newcastle University; Campus for Ageing and Vitality; Newcastle upon Tyne, UK
&
Rudolf Jaenisch Whitehead Institute for Biomedical Research; Massachusetts Institute of Technology; Cambridge, MA USA; Skolkovo Institute of Science and Technology (Skoltech); Skolkovo, Moscow Region, RussiaCorrespondence[email protected] [email protected]
Pages 1137-1140 | Received 12 Mar 2014, Accepted 21 Mar 2014, Published online: 10 Apr 2014

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Sheng Dai, Andrés E. Dulcey, Xin Hu, Christopher A. Wassif, Forbes D. Porter, Christopher P. Austin, Daniel S. Ory, Juan Marugan & Wei Zheng. (2017) Methyl-β-cyclodextrin restores impaired autophagy flux in Niemann-Pick C1-deficient cells through activation of AMPK. Autophagy 13:8, pages 1435-1451.
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Articles from other publishers (16)

Hadas Ben-Zvi, Tatiana Rabinski, Rivka Ofir, Smadar Cohen & Gad D. Vatine. (2022) PLEKHM2 Loss of Function Impairs the Activity of iPSC-Derived Neurons via Regulation of Autophagic Flux. International Journal of Molecular Sciences 23:24, pages 16092.
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Wenqi Dong, Gaoyan Wang, Jiajia Feng, Pei Li, Rui Wang, Hao Lu, Wenjia Lu, Chenchen Wang, Xiangru Wang, Huanchun Chen, Yaozu Xiang & Chen Tan. (2022) MiR-25 blunts autophagy and promotes the survival of Mycobacterium tuberculosis by regulating NPC1. iScience 25:5, pages 104279.
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Daniela Castiblanco, Jesse A. Rudd-Schmidt, Tahereh Noori, Vivien R. SuttonYa Hui Hung, Thijs W. H. Flinsenberg, Adrian W. Hodel, Neil D. Young, Nicholas Smith, Drago BratkovicHeidi PetersMark Walterfang, Joseph A. Trapani, Amelia J. Brennan & Ilia Voskoboinik. (2022) Severely impaired CTL killing is a feature of the neurological disorder Niemann-Pick disease type C1. Blood 139:12, pages 1833-1849.
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Irmgard Tegeder & Donat Kögel. (2021) When lipid homeostasis runs havoc: Lipotoxicity links lysosomal dysfunction to autophagy. Matrix Biology 100-101, pages 99-117.
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Jason Newton, Elisa N.D. Palladino, Cynthia Weigel, Michael Maceyka, Markus H. Gräler, Can E. Senkal, Ricardo D. Enriz, Pavlina Marvanova, Josef Jampilek, Santiago Lima, Sheldon Milstien & Sarah Spiegel. (2020) Targeting defective sphingosine kinase 1 in Niemann–Pick type C disease with an activator mitigates cholesterol accumulation. Journal of Biological Chemistry 295:27, pages 9121-9133.
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Jason Newton, Sheldon Milstien & Sarah Spiegel. (2018) Niemann-Pick type C disease: The atypical sphingolipidosis. Advances in Biological Regulation 70, pages 82-88.
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Antony Cougnoux, Miyad Movassaghi, Jaqueline A. Picache, James R. Iben, Fatemeh Navid, Alexander Salman, Kyle Martin, Nicole Y. Farhat, Celine Cluzeau, Wei-Chia Tseng, Kathryn Burkert, Caitlin Sojka, Christopher A. Wassif, Niamh X. Cawley, Richard Bonnet & Forbes D. Porter. (2018) Gastrointestinal Tract Pathology in a BALB/c Niemann–Pick Disease Type C1 Null Mouse Model. Digestive Diseases and Sciences 63:4, pages 870-880.
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Elena SeranovaKyle J. ConnollyMalgorzata ZatykaTatiana R. Rosenstock, Timothy BarrettRichard I. TuxworthSovan Sarkar. (2017) Dysregulation of autophagy as a common mechanism in lysosomal storage diseases. Essays in Biochemistry 61:6, pages 733-749.
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Matthis Synofzik & Rebecca Schüle. (2017) Overcoming the divide between ataxias and spastic paraplegias: Shared phenotypes, genes, and pathways. Movement Disorders 32:3, pages 332-345.
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Megan M. Young, Yoshinori Takahashi, Todd E. Fox, Jong K. Yun, Mark Kester & Hong-Gang Wang. (2016) Sphingosine Kinase 1 Cooperates with Autophagy to Maintain Endocytic Membrane Trafficking. Cell Reports 17:6, pages 1532-1545.
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Anna Brown, Siddharth Patel, Carl Ward, Anna Lorenz, Mauren Ortiz, Allison DuRoss, Fabian Wieghardt, Amanda Esch, Elsje G. Otten, Laura M. Heiser, Viktor I. Korolchuk, Conroy Sun, Sovan Sarkar & Gaurav Sahay. (2016) PEG-lipid micelles enable cholesterol efflux in Niemann-Pick Type C1 disease-based lysosomal storage disorder. Scientific Reports 6:1.
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Carl Ward, Nuria Martinez-Lopez, Elsje G. Otten, Bernadette Carroll, Dorothea Maetzel, Rajat Singh, Sovan Sarkar & Viktor I. Korolchuk. (2016) Autophagy, lipophagy and lysosomal lipid storage disorders. Biochimica et Biophysica Acta (BBA) - Molecular and Cell Biology of Lipids 1861:4, pages 269-284.
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Roy Karnati, Venu Talla, Katherine Peterson & Gordon W. Laurie. (2016) Lacritin and other autophagy associated proteins in ocular surface health. Experimental Eye Research 144, pages 4-13.
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Darius Ebrahimi-Fakhari, Afshin Saffari, Lara Wahlster, Jenny Lu, Susan Byrne, Georg F. Hoffmann, Heinz Jungbluth & Mustafa Sahin. (2016) Congenital disorders of autophagy: an emerging novel class of inborn errors of neuro-metabolism. Brain 139:2, pages 317-337.
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Jacob L. Mercer, Joseph P. Argus, Donna M. Crabtree, Melissa M. Keenan, Moses Q. Wilks, Jen-Tsan Ashley Chi, Steven J. Bensinger, Catherine P. Lavau & Daniel S. Wechsler. (2015) Modulation of PICALM Levels Perturbs Cellular Cholesterol Homeostasis. PLOS ONE 10:6, pages e0129776.
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Marie T. Vanier. (2014) Complex lipid trafficking in Niemann-Pick disease type C. Journal of Inherited Metabolic Disease 38:1, pages 187-199.
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