A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1
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Title
A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1
Authors
Keywords
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Journal
BMC BIOLOGY
Volume 19, Issue 1, Pages -
Publisher
Springer Science and Business Media LLC
Online
2021-10-01
DOI
10.1186/s12915-021-01133-x
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Note: Only part of the references are listed.- Animal models for Niemann-Pick type C: implications for drug discovery & development
- (2019) Cathrine K. Fog et al. Expert Opinion on Drug Discovery
- Cytosolic glucosylceramide regulates endolysosomal function in Niemann-Pick type C disease
- (2019) Simon Wheeler et al. NEUROBIOLOGY OF DISEASE
- Neuronal Soma-Derived Degradative Lysosomes Are Continuously Delivered to Distal Axons to Maintain Local Degradation Capacity
- (2019) Tamar Farfel-Becker et al. Cell Reports
- CRISPR Interference-Based Platform for Multimodal Genetic Screens in Human iPSC-Derived Neurons
- (2019) Ruilin Tian et al. NEURON
- Axonal transport and maturation of lysosomes
- (2018) Shawn M Ferguson CURRENT OPINION IN NEUROBIOLOGY
- Lysosome trafficking and signaling in health and neurodegenerative diseases
- (2018) Pearl P.Y. Lie et al. NEUROBIOLOGY OF DISEASE
- Modeling Niemann-Pick disease type C1 in zebrafish: a robust platform forin vivoscreening of candidate therapeutic compounds
- (2018) Wei-Chia Tseng et al. Disease Models & Mechanisms
- Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial
- (2017) Daniel S Ory et al. LANCET
- Scalable Production of iPSC-Derived Human Neurons to Identify Tau-Lowering Compounds by High-Content Screening
- (2017) Chao Wang et al. Stem Cell Reports
- Neuronal energy consumption: biophysics, efficiency and evolution
- (2016) Jeremy E Niven CURRENT OPINION IN NEUROBIOLOGY
- Reversal of Pathologic Lipid Accumulation in NPC1-Deficient Neurons by Drug-Promoted Release of LAMP1-Coated Lamellar Inclusions
- (2016) Valérie Demais et al. JOURNAL OF NEUROSCIENCE
- Metabolic reprogramming during neuronal differentiation from aerobic glycolysis to neuronal oxidative phosphorylation
- (2016) Xinde Zheng et al. eLife
- A Murine Niemann-Pick C1 I1061T Knock-In Model Recapitulates the Pathological Features of the Most Prevalent Human Disease Allele
- (2015) M. Praggastis et al. JOURNAL OF NEUROSCIENCE
- Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease
- (2015) Charles H. Vite et al. Science Translational Medicine
- Improved vectors and genome-wide libraries for CRISPR screening
- (2014) Neville E Sanjana et al. NATURE METHODS
- Easy quantitative assessment of genome editing by sequence trace decomposition
- (2014) Eva K. Brinkman et al. NUCLEIC ACIDS RESEARCH
- Visualization of cholesterol deposits in lysosomes of Niemann-Pick type C fibroblasts using recombinant perfringolysin O
- (2014) Katarzyna Kwiatkowska et al. Orphanet Journal of Rare Diseases
- Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling
- (2013) Martin Fan et al. JOURNAL OF LIPID RESEARCH
- Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells
- (2013) Ophir Shalem et al. SCIENCE
- Therapeutic potential of cyclodextrins in the treatment of Niemann–Pick type C disease
- (2012) Benny Liu Clinical Lipidology
- Oxidative stress in Niemann–Pick disease, type C
- (2010) Rao Fu et al. MOLECULAR GENETICS AND METABOLISM
- Niemann-Pick disease type C
- (2010) Marie T Vanier Orphanet Journal of Rare Diseases
- Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression
- (2009) Cristin D. Davidson et al. PLoS One
- Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1-/- mouse
- (2009) B. Liu et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Understanding the Warburg Effect: The Metabolic Requirements of Cell Proliferation
- (2009) M. G. Vander Heiden et al. SCIENCE
- Advances in measuring cellular bioenergetics using extracellular flux
- (2008) David A. Ferrick et al. DRUG DISCOVERY TODAY
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