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1. Full Text Molecular mechanisms of endolysosomal Ca 2+ signalling in health and disease
by Morgan, Anthony J and Platt, Frances M and Lloyd-Evans, Emyr and Galione, Antony
Biochemical Journal, ISSN 0264-6021, 11/2011, Volume 439, Issue 3, pp. 349 - 374
Calcium store | Endolysosome | Nicotinic acid adenine dinucleotide phosphate (NAADP) | ATPase | Two-pore channel (TPC)
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2. Lysosomes and lysosomal diseases
by Platt, Frances M and Platt, Nick
2015, First edition., Methods in cell biology, ISBN 9780128000793, Volume 126
Lysosomes | Phagosomes | Lysosomal storage diseases | Phagocytosis
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3. Lysosomal disorders of the brain
: recent advances in molecular and cellular pathogenesis and treatment
by Platt, Frances M and Walkley, Steven U
2004, ISBN 0198508786, xxvii. 447
Lysosomal storage diseases are inherited metabolic disorders characterized by severe pathology, typically involving the brain. Although individually rare, they... 
Brain | Lysosomal storage diseases | Treatment | Metabolism | Disorders | neuroscience | Lysosomal storage disorders - Treatment
Book
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4. Full Text Miglustat
: A Viewpoint by Frances M. Platt and Terry D. Butters
by Platt, Frances M and Butters, Terry D
Drugs, ISSN 0012-6667, 11/2003, Volume 63, Issue 22, pp. 2435 - 2435
Pharmacotherapy | Internal Medicine | Medicine & Public Health | Pharmacology/Toxicology
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5. Full Text Emptying the stores: lysosomal diseases and therapeutic strategies
by Platt, Frances M
Nature reviews. Drug discovery, 11/2017
Lysosomal storage disorders (LSDs) - designated as 'orphan' diseases - are inborn errors of metabolism caused by defects in genes that encode proteins involved... 
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6. Full Text Front Cover: Synthesis and Study of Multifunctional Cyclodextrin–Deferasirox Hybrids (ChemMedChem 16/2019)
by Gascon, Jose Miguel and Oliveri, Valentina and McGown, Andrew and Kaya, Ecem and Chen, Yu‐Lin and Austin, Carol and Walker, Martin and Platt, Frances M and Vecchio, Graziella and Spencer, John
ChemMedChem, ISSN 1860-7179, 08/2019, Volume 14, Issue 16, pp. 1466 - 1466
The Front Cover depicts two horses (taken by the lead author J.S.) that look like they are “joined at the hips.” Although they look “connected,” each horse... 
antioxidant | Niemann–Pick | protein aggregation | deferasirox | iron complexes
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7. Full Text Lysosomes and Lysosomal Diseases
by Platt, Frances M and Platt, Nick
2015, ISBN 9780128000793, 451
This new volume of Methods in Cell Biology looks at methods for lysosomes and lysosomal diseases. Chapters focus upon practical experimental protocols to guide... 
Lysosomes | Biology, life sciences | Lysosomal storage diseases
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8. Full Text Lysosomal storage disorders: The cellular impact of lysosomal dysfunction
by Platt, Frances M and Boland, Barry and van der Spoel, Aarnoud C
Journal of Cell Biology, ISSN 0021-9525, 11/2012, Volume 199, Issue 5, pp. 723 - 734
Lysosomal storage diseases (LSDs) are a family of disorders that result from inherited gene mutations that perturb lysosomal homeostasis. LSDs mainly stem from... 
LATE ENDOSOMES | CHAPERONE-MEDIATED AUTOPHAGY | CHOLESTEROL ACCUMULATION | CALCIUM HOMEOSTASIS | MOUSE MODEL | MITOCHONDRIAL ABERRATIONS | DISEASE | GENE-THERAPY | SALVAGE PATHWAY | ENZYME REPLACEMENT THERAPY | CELL BIOLOGY | Gene mutations | Lysosomes | Physiological aspects | Cytogenetics | Homeostasis | Genetic aspects | Research | Health aspects | Proteins | Mutation | Cellular biology | Metabolic disorders | Reviews
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9. Full Text Miglustat: A Viewpoint by Frances M. Platt and Terry D. Butters
by Platt F.M and Butters T.D
Drugs, ISSN 0012-6667, 2003, Volume 63, Issue 22, pp. 2425 - 2426
Glucosylceramide synthase inhibitors, general | Miglustat, general | Research and development | Adis Drug Profiles | Gaucher's disease
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10. Full Text Lysosomal Ca.sup.2+ homeostasis: Role in pathogenesis of lysosomal storage diseases
by Lloyd-Evans, Emyr and Platt, Frances M
Cell Calcium, ISSN 0143-4160, 08/2011, Volume 50, Issue 2
Disrupted cellular Ca.sup.2+ signaling is believed to play a role in a number of human diseases including lysosomal storage diseases (LSD). LSDs are a group of... 
Proteins | Metabolism, Inborn errors of
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11. Full Text RIPK3 as a potential therapeutic target for Gaucher's disease
by Vitner, Einat B and Salomon, Ran and Farfel-Becker, Tamar and Meshcheriakova, Anna and Ali, Mohammad and Klein, Andrés D and Platt, Frances M and Cox, Timothy M and Futerman, Anthony H
Nature Medicine, ISSN 1078-8956, 02/2014, Volume 20, Issue 2, pp. 204 - 208
Gaucher's disease (GD), an inherited metabolic disorder caused by mutations in the glucocerebrosidase gene (GBA), is the most common lysosomal storage... 
MEDICINE, RESEARCH & EXPERIMENTAL | APOPTOSIS | NECROPTOSIS | DOMAIN KINASE RIP | BIOCHEMISTRY & MOLECULAR BIOLOGY | INDUCED INFLAMMATION | CELL-DEATH | CELL BIOLOGY | RIPOPTOSOME | NECROSIS | INHIBITION | MOUSE MODEL | NEURONOPATHIC FORMS | Receptor-Interacting Protein Serine-Threonine Kinases - metabolism | Liver - pathology | Gaucher Disease - metabolism | Motor Skills - physiology | Mice, Inbred C57BL | Fluoresceins | Gaucher Disease - pathology | Blotting, Western | Mice, Knockout | Animals | Gaucher Disease - genetics | Cell Death - physiology | Brain - pathology | Mice | Receptor-Interacting Protein Serine-Threonine Kinases - deficiency | Physiological aspects | Medicine, Experimental | Medical research | Care and treatment | Protein kinases | Gaucher's disease | Proteins | Pharmacology | Genetic disorders | Drug therapy | Kinases | Metabolic disorders | Index Medicus
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12. Full Text Urinary excretion and metabolism of miglustat and valproate in patients with Niemann--Pick type C1 disease: One- and two-dimensional solution-state .sup.1H NMR studies
by Probert, Fay and Ruiz-Rodado, Victor and Zhang, Xiaoyu and Vruchte, Danielle te and Claridge, Tim D.W and Edgar, Mark and Tocchio, Anna Zonato and Lachmann, Robin H and Platt, Frances M and Grootveld, Martin
Journal of Pharmaceutical and Biomedical Analysis, ISSN 0731-7085, 01/2016, Volume 117, p. 276
To access, purchase, authenticate, or subscribe to the full-text of this article, please visit this link: http://dx.doi.org/10.1016/j.jpba.2015.08.011... 
Urine | Metabolites | Analysis | Physiological aspects
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13. Full Text Urinary excretion and metabolism of miglustat and valproate in patients with Niemann–Pick type C1 disease: One- and two-dimensional solution-state 1 H NMR studies
by Probert, Fay and Ruiz-Rodado, Victor and Zhang, Xiaoyu and Vruchte, Danielle te and Claridge, Tim D.W and Edgar, Mark and Tocchio, Anna Zonato and Lachmann, Robin H and Platt, Frances M and Grootveld, Martin
Journal of Pharmaceutical and Biomedical Analysis, ISSN 0731-7085, 01/2016, Volume 117, pp. 276 - 288
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14. Full Text Lysosomal Ca2+ homeostasis: Role in pathogenesis of lysosomal storage diseases
by Lloyd-Evans, Emyr and Platt, Frances M
Cell Calcium, ISSN 0143-4160, 2011, Volume 50, Issue 2, pp. 200 - 205
Abstract Disrupted cellular Ca2+ signaling is believed to play a role in a number of human diseases including lysosomal storage diseases (LSD). LSDs are a... 
Advanced Basic Science | Mucolipidosis type IV | NAADP | NPC1 | MLIV | Lysosomal calcium | Lysosomal storage disease | Niemann-Pick | PROTEIN-KINASE-C | MUCOLIPIDOSIS TYPE-IV | CATION CHANNEL | ACIDIC ORGANELLES | RELEASE CHANNEL | PLASMA-MEMBRANE | CELL BIOLOGY | SPHINGOLIPID METABOLISM | CALCIUM-RELEASE | CHEDIAK-HIGASHI-SYNDROME | 2-PORE CHANNELS
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15. Full Text Autophagy Induction and Autophagosome Clearance in Neurons: Relationship to Autophagic Pathology in Alzheimer's Disease
by Boland, Barry and Kumar, Asok and Lee, Sooyeon and Platt, Frances M and Wegiel, Jerzy and Yu, W. Haung and Nixon, Ralph A
Journal of Neuroscience, ISSN 0270-6474, 07/2008, Volume 28, Issue 27, pp. 6926 - 6937
Macroautophagy, a major pathway for organelle and protein turnover, has been implicated in the neurodegeneration of Alzheimer's disease (AD). The basis for the... 
Neuroprotection | Neurotoxicity | Neuron | Storage | Neuropathology | Lysosome | Neuron death | Aging | Neuronal death | Alzheimer | Autophagy | Culture | NEURODEGENERATIVE DISEASE | RAT-LIVER | APOPTOSIS | autophagy | neuron | VINBLASTINE | storage | MACROAUTOPHAGY | MAMMALIAN-CELLS | NEUROSCIENCES | neuron death | TRANSPORT | neurotoxicity | culture | neuronal death | neuroprotection | MONITORING AUTOPHAGY | DEGRADATION | MICE | aging | lysosome | neuropathology | Protein Kinases - metabolism | Neurons - pathology | Microtubule-Associated Proteins - metabolism | Tubulin Modulators - pharmacology | Peptide Hydrolases - drug effects | Autophagy - physiology | Alzheimer Disease - pathology | Brain - metabolism | Lysosomes - metabolism | Neurons - ultrastructure | Neurites - ultrastructure | Brain - ultrastructure | Lysosomes - pathology | Phagosomes - ultrastructure | Neurons - metabolism | Peptide Hydrolases - metabolism | Animals, Newborn | Alzheimer Disease - physiopathology | Protein Kinases - drug effects | Phagosomes - metabolism | Cells, Cultured | Rats | Neurites - metabolism | Phagosomes - pathology | Rats, Sprague-Dawley | Lysosomes - ultrastructure | Neurites - pathology | Animals | Cathepsins - metabolism | Alzheimer Disease - metabolism | Brain - pathology | TOR Serine-Threonine Kinases | Vinblastine - pharmacology | Index Medicus | Aging [Ageing] | Neuron Death
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16. Full Text Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium
by Sillence, Daniel J and Morgan, Anthony J and Smith, David A and Churchill, Grant C and Galione, Antony and Platt, Frances M and Schuchman, Edward H and Elliot-Smith, Elena and Lloyd-Evans, Emyr and He, Xingxuan
Nature Medicine, ISSN 1078-8956, 11/2008, Volume 14, Issue 11, pp. 1247 - 1255
Niemann-Pick type C1 (NPC1) disease is a neurodegenerative lysosomal storage disorder caused by mutations in the acidic compartment (which we define as the... 
MEDICINE, RESEARCH & EXPERIMENTAL | CELLS | NPC1 PROTEIN | CHOLESTEROL | BIOCHEMISTRY & MOLECULAR BIOLOGY | MODEL | CERAMIDE | CELL BIOLOGY | INTRACELLULAR CA2 | METABOLISM | STORES | MICE | BINDING | Niemann-Pick Disease, Type C - drug therapy | Membrane Glycoproteins - metabolism | Calcium - metabolism | Humans | Homeostasis | Niemann-Pick Disease, Type C - classification | Niemann-Pick Disease, Type C - metabolism | Lysosomes - metabolism | Sphingosine - metabolism | Cell Line | Curcumin - therapeutic use | Cholesterol - metabolism | Mutation - genetics | Membrane Glycoproteins - genetics | Mice, Knockout | Proteins - genetics | Carrier Proteins - genetics | Phenotype | Animals | Carrier Proteins - metabolism | Proteins - metabolism | Acids | Glycosphingolipids - metabolism | Mice | Niemann-Pick Disease, Type C - genetics | Niemann-Pick disease | Care and treatment | Gene mutations | Calcium metabolism disorders | Causes of | Genetic aspects | Research | Health aspects | Risk factors | Genotype & phenotype | Pathology | Mutation | Cellular biology | Neurological disorders | Index Medicus
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17. Full Text Lysosomal Ca 2+ homeostasis: Role in pathogenesis of lysosomal storage diseases
by Lloyd-Evans, Emyr and Platt, Frances M
Cell Calcium, ISSN 0143-4160, 2011, Volume 50, Issue 2, pp. 200 - 205
Disrupted cellular Ca signaling is believed to play a role in a number of human diseases including lysosomal storage diseases (LSD). LSDs are a group of ∼50... 
Mucolipidosis type IV | NAADP | NPC1 | MLIV | Lysosomal calcium | Lysosomal storage disease | Niemann-Pick
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18. Full Text Expression of Ca2+‐permeable two‐pore channels rescues NAADP signalling in TPC‐deficient cells
by Ruas, Margarida and Davis, Lianne C and Chen, Cheng‐Chang and Morgan, Anthony J and Chuang, Kai‐Ting and Walseth, Timothy F and Grimm, Christian and Garnham, Clive and Powell, Trevor and Platt, Nick and Platt, Frances M and Biel, Martin and Wahl‐Schott, Christian and Parrington, John and Galione, Antony
The EMBO Journal, ISSN 0261-4189, 07/2015, Volume 34, Issue 13, pp. 1743 - 1758
The second messenger NAADP triggers Ca 2+ release from endo‐lysosomes. Although two‐pore channels ( TPC s) have been proposed to be regulated by NAADP , recent... 
endo‐lysosome | NAADP | electrophysiology | TPC | ACTIVATION | ISOFORMS | ORGANELLES | BIOCHEMISTRY & MOLECULAR BIOLOGY | endo-lysosome | GRANULES | CELL BIOLOGY | Ca2 | CALCIUM | ION-CHANNEL | PROTEINS | Lysosomes - drug effects | Calcium Channels - metabolism | Calcium - metabolism | Cells, Cultured | Lysosomes - physiology | NADP - pharmacology | Mice, Knockout | Evoked Potentials - drug effects | Animals | Protein Isoforms - metabolism | Signal Transduction - drug effects | Calcium Signaling - drug effects | Gene Expression - physiology | NADP - analogs & derivatives | Mice | NADP - metabolism | Calcium Signaling - genetics | Calcium Channels - genetics | Hydrogen-Ion Concentration | Protein Isoforms - genetics | Proteins | Biochemistry | Cellular biology | Molecular biology | Index Medicus
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19. Full Text Chemoenzymatic Synthesis of a Phosphorylated Glycoprotein
by Pragya Priyanka and Thomas B Parsons and Antonia Miller and Frances M Platt and Antony J Fairbanks
Angewandte Chemie, ISSN 0044-8249, 04/2016, Volume 128, Issue 16, p. 5142
  The majority of lysosomal enzymes are targeted to the lysosome by post-translational tagging with N-glycans terminating in mannose-6-phosphate (M6P)... 
Enzymes | Chemical synthesis
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20. Full Text Modulation of Human Natural Killer T Cell Ligands on TLR-Mediated Antigen-Presenting Cell Activation
by Mariolina Salio and Anneliese O. Speak and Dawn Shepherd and Paolo Polzella and Petr A. Illarionov and Natacha Veerapen and Gurdyal S. Besra and Frances M. Platt and Vincenzo Cerundolo
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 12/2007, Volume 104, Issue 51, pp. 20490 - 20495
Invariant natural killer T (iNKT) cells are a subset of nonconventional T cells recognizing endogenous and/or exogenous glycolipid antigens in the context of... 
T lymphocytes | Molecules | Antigens | Natural killer T cells | Secretion | Cell lines | Antibodies | Ligands | Lipids | T cell antigen receptors | Innate immunity | Glycosphingolipids | NKT cells | APC | INFECTIONS | PLASMACYTOID-DENDRITIC-CELLS | RECOGNITION | MULTIDISCIPLINARY SCIENCES | ISOGLOBOTRIHEXOSYLCERAMIDE | CD1D | DEFICIENCY | innate immunity | glycosphingolipids | IN-VIVO | EXPRESSION | T-Lymphocyte Subsets - immunology | Antigens, CD1 - immunology | Lymphocyte Activation | Dendritic Cells - immunology | Humans | Cell Line, Tumor | Glycosphingolipids - metabolism | Killer Cells, Natural - immunology | Receptors, Antigen, T-Cell - agonists | Interferon-gamma - metabolism | Antigen-Presenting Cells - immunology | Antigen presenting cells | Sphingolipids | T cells | Properties | Ligands (Biochemistry) | Immunological research | Antigen presentation | T cell receptors | Maturation | Index Medicus | Biological Sciences
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