David A Smith

发表

Anti-TNF therapy for inflammatory bowel disease in patients with neurodegenerative Niemann-Pick disease Type C [version 1; peer review: 1 approved]

H. Uhlig, F. Platt, L. Peyrin-Biroulet, 2022 .

Anti-TNF therapy for inflammatory bowel disease in patients with neurodegenerative Niemann-Pick disease Type C [version 1; peer review: awaiting peer review]

H. Uhlig, F. Platt, L. Peyrin-Biroulet, 2022 .

Beneficial effects of anti-inflammatory therapy in a mouse model of Niemann-Pick disease type C1

F. Platt, Kerri-Lee Wallom, David A Smith, 2009, Neurobiology of Disease.

Relative acidic compartment volume as a lysosomal storage disorder-associated biomarker.

Anneliese O. Speak, A. Speak, H. Runz, 2014, The Journal of clinical investigation.

Improved neuroprotection using miglustat, curcumin and ibuprofen as a triple combination therapy in Niemann–Pick disease type C1 mice

F. Platt, Kerri-Lee Wallom, David A Smith, 2014, Neurobiology of Disease.

Immune dysfunction in Niemann‐Pick disease type C

Anneliese O. Speak, A. Speak, F. Platt, 2015, Journal of neurochemistry.

Anti-TNF therapy for inflammatory bowel disease in patients with neurodegenerative Niemann-Pick disease Type C

H. Uhlig, F. Platt, L. Peyrin-Biroulet, 2022, Wellcome open research.

An Inducible Mouse Model of Late Onset Tay–Sachs Disease

V. Perry, R. Dwek, M. Cortina-Borja, 2002, Neurobiology of Disease.

1H NMR-Linked Metabolomics Analysis of Liver from a Mouse Model of NP-C1 Disease.

F. Platt, M. Grootveld, V. Ruiz-Rodado, 2016, Journal of proteome research.

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann–Pick type C1 disease

E. Henckaerts, F. Platt, A. Rahim, 2018, Human molecular genetics.

Beneficial Effects of Acetyl-DL-Leucine (ADLL) in a Mouse Model of Sandhoff Disease

M. Strupp, F. Platt, David A Smith, 2020, Journal of clinical medicine.

Altered distribution and function of natural killer cells in murine and human Niemann-Pick disease type C1.

Anneliese O. Speak, A. Speak, A. Galione, 2014, Blood.

Critical role of iron in the pathogenesis of the murine gangliosidoses

F. Platt, T. Cox, David A Smith, 2009, Neurobiology of Disease.

Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis.

Anneliese O. Speak, A. Speak, R. Dwek, 2008, Molecular genetics and metabolism.

NSAIDs increase survival in the Sandhoff disease mouse: Synergy with N‐butyldeoxynojirimycin

R. Dwek, F. Platt, M. C. Borja, 2004, Annals of neurology.

Differential response of the liver to bile acid treatment in a mouse model of Niemann-Pick disease type C

F. Platt, David A Smith, Mylene Huebecker, 2017, Wellcome open research.

Differential response of the liver to bile acid treatment in a mouse model of Niemann-Pick disease type C [ version 1

F. Platt, David A Smith, Lauren Morris, 2019 .

Invariant natural killer T cells are not affected by lysosomal storage in patients with Niemann‐Pick disease type C

Anneliese O. Speak, A. Speak, G. Besra, 2012, European journal of immunology.

Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium

A. Galione, G. Churchill, F. Platt, 2008, Nature Medicine.

Improved outcome of N-butyldeoxygalactonojirimycin-mediated substrate reduction therapy in a mouse model of Sandhoff disease

R. Dwek, F. Platt, M. C. Borja, 2004, Neurobiology of Disease.

Heat shock protein amplification improves cerebellar myelination in the Npc1nih mouse model

F. Platt, T. Kirkegaard, David A Smith, 2022, EBioMedicine.

Early glial activation, synaptic changes and axonal pathology in the thalamocortical system of Niemann–Pick type C1 mice

J. Cooper, F. Platt, David A Smith, 2012, Neurobiology of Disease.

Imaging of changes in copper trafficking and redistribution in a mouse model of Niemann-Pick C disease using positron emission tomography

J. Spencer, F. Platt, R. Purchase, 2019, BioMetals.

Macroautophagy Is Not Directly Involved in the Metabolism of Amyloid Precursor Protein*

D. Walsh, F. Platt, David A Smith, 2010, The Journal of Biological Chemistry.

Combined Anti-inflammatory and Neuroprotective Treatments Have the Potential to Impact Disease Phenotypes in Cln3−/− Mice

J. Cooper, F. Platt, A. Najafi, 2019, Front. Neurol..

Defective Cytochrome P450-Catalysed Drug Metabolism in Niemann-Pick Type C Disease

W. Pavan, C. Vite, W. Figg, 2016, PloS one.

Neural Stem Cell Transplantation Benefits a Monogenic Neurometabolic Disorder During the Symptomatic Phase of Disease

E. Snyder, N. Sibson, F. Platt, 2009, Stem cells.

Development of gene therapy for Niemann-Pick Type C disease

E. Henckaerts, F. Platt, A. Rahim, 2015 .

Defective iron homeostasis and hematological abnormalities in Niemann-Pick disease type C1

F. Platt, F. Porter, A. Abdul-Sada, 2022, Wellcome open research.

Defective platelet function in Niemann‐Pick disease type C1

Anneliese O. Speak, A. Speak, A. Galione, 2020, JIMD reports.

Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses

D. Begley, A. Hinsby, M. Jäättelä, 2016, Science Translational Medicine.

Acetyl-leucine slows disease progression in lysosomal storage disorders

M. Strupp, J. Spencer, M. Cortina-Borja, 2020, bioRxiv.

Heat shock protein-based therapy for sphingolipidoses

D. Begley, A. Hinsby, M. Jäättelä, 2017 .

Circadian profiling in two mouse models of lysosomal storage disorders; Niemann Pick type-C and Sandhoff disease

L. Brown, P. Oliver, S. Peirson, 2016, Behavioural Brain Research SreeTestContent1.

A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease

A. Rahim, David A Smith, Claire Smith, 2023, Cells.

Critical role of iron in the pathogenesis of the murine gangliosidoses

F. Platt, T. Cox, David A Smith, 2009, Neurobiology of Disease.