Pluripotent stem cells in disease modelling and drug discovery

Y Avior, I Sagi, N Benvenisty - Nature reviews Molecular cell biology, 2016 - nature.com
Experimental modelling of human disorders enables the definition of the cellular and
molecular mechanisms underlying diseases and the development of therapies for treating …

[HTML][HTML] Autophagy, lipophagy and lysosomal lipid storage disorders

C Ward, N Martinez-Lopez, EG Otten, B Carroll… - … et Biophysica Acta (BBA …, 2016 - Elsevier
Autophagy is a catabolic process with an essential function in the maintenance of cellular
and tissue homeostasis. It is primarily recognised for its role in the degradation of …

Dysregulation of autophagy as a common mechanism in lysosomal storage diseases

E Seranova, KJ Connolly, M Zatyka… - Essays in …, 2017 - portlandpress.com
The lysosome plays a pivotal role between catabolic and anabolic processes as the nexus
for signalling pathways responsive to a variety of factors, such as growth, nutrient …

Sphingosine 1-phosphate and sphingosine kinases in health and disease: Recent advances

S Pyne, DR Adams, NJ Pyne - Progress in lipid research, 2016 - Elsevier
Sphingosine kinases (isoforms SK1 and SK2) catalyse the formation of a bioactive lipid,
sphingosine 1-phosphate (S1P). S1P is a well-established ligand of a family of five S1P …

[HTML][HTML] The Niemann-Pick type diseases–A synopsis of inborn errors in sphingolipid and cholesterol metabolism

FW Pfrieger - Progress in lipid research, 2023 - Elsevier
Disturbances of lipid homeostasis in cells provoke human diseases. The elucidation of the
underlying mechanisms and the development of efficient therapies represent formidable …

[HTML][HTML] Enrichment of NPC1-deficient cells with the lipid LBPA stimulates autophagy, improves lysosomal function, and reduces cholesterol storage

O Ilnytska, K Lai, K Gorshkov, ML Schultz… - Journal of Biological …, 2021 - ASBMB
Niemann–Pick C (NPC) is an autosomal recessive disorder characterized by mutations in
the NPC1 or NPC2 genes encoding endolysosomal lipid transport proteins, leading to …

[HTML][HTML] Neuronal SphK1 acetylates COX2 and contributes to pathogenesis in a model of Alzheimer's Disease

JY Lee, SH Han, MH Park, B Baek, IS Song… - Nature …, 2018 - nature.com
Although many reports have revealed the importance of defective microglia-mediated
amyloid β phagocytosis in Alzheimer's disease (AD), the underlying mechanism remains to …

Niemann-Pick type C disease: The atypical sphingolipidosis

J Newton, S Milstien, S Spiegel - Advances in biological regulation, 2018 - Elsevier
Abstract Niemann-Pick type C (NPC) disease is a lysosomal storage disorder resulting from
mutations in either the NPC1 (95%) or NPC2 (5%) genes. NPC typically presents in …

[HTML][HTML] Lysosomal and mitochondrial liaisons in Niemann-Pick disease

S Torres, E Balboa, S Zanlungo, C Enrich… - Frontiers in …, 2017 - frontiersin.org
Lysosomal storage disorders (LSD) are characterized by the accumulation of diverse lipid
species in lysosomes. Niemann-Pick type A/B (NPA/B) and type C diseases Niemann-Pick …

Effect of lysosomotropic molecules on cellular homeostasis

OF Kuzu, M Toprak, MA Noory, GP Robertson - Pharmacological research, 2017 - Elsevier
Weak bases that readily penetrate through the lipid bilayer and accumulate inside the acidic
organelles are known as lysosomotropic molecules. Many lysosomotropic compounds …