Head of Department
- Professor of Biochemistry and Pharmacology
Professor Platt obtained a BSc in Zoology at Imperial College University of London and a PhD in animal physiology from the University of Bath. She was a post-doctoral fellow at Washington University Medical School in St Louis, USA. She returned to the UK in 1989 (to the Biochemistry Department, University of Oxford) where she focused on how the abnormal accumulation of glycosphingolipids results in pathology in lysosomal storage diseases.
She was a Lister Institute Senior Research Fellow from 1996-2002. A major focus of her work has been on the development of substrate reduction therapy (SRT) to treat several of these disorders. Proof of principle of SRT was demonstrated in mouse models of these primarily neurodegenerative diseases. Dr Platt’s research, in collaboration with Dr Terry Butters, has led to the development of the approved drug miglustat/Zavesca for glycosphingolipid storage disease therapy.
Her current interests focus on the cell biology and pathobiology of glycosphingolipids and on the development of novel therapies for treating diseases resulting from defects in gycolipid metabolism and lysosomal dysfunction.
She moved to the Department of Pharmacology in April 2006 and was elected a fellow of the Academy of Medical Sciences in 2011.
Key Research Areas:
- Lysosomal storage disorders, pathogenesis and therapy
- The effects of lysosomal storage on the immune system
- Development of biomarkers for monitoring storage disease patients
- Lysosomal dysfunction in more common diseases
A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease
Hughes MP. et al, (2023), Cells, 12, 1619 - 1619
Grafting of Cyclodextrin to Theranostic Nanoparticles Im-proves Blood-Brain Barrier Model Crossing
PLATT F., (2023), Biomolecules
A Mouse Systems Genetics Approach Reveals Common and Uncommon Genetic Modifiers of Hepatic Lysosomal Enzyme Activities and Glycosphingolipids.
Durán A. et al, (2023), Int J Mol Sci, 24
Heat shock protein amplification improves cerebellar myelination in the Npc1nih mouse model.
Gray J. et al, (2022), EBioMedicine, 86
Defective iron homeostasis and hematological abnormalities in Niemann-Pick disease type C1
Chen OCW. et al, (2022), Wellcome Open Research, 7, 267 - 267
Inhibition of the Niemann-Pick C1 protein is a conserved feature of multiple strains of pathogenic mycobacteria.
Weng Y. et al, (2022), Nat Commun, 13
Unravelling the role of acidic organelles in Goat Atrial Fibrillation
Ayagama T. et al, (2022), ACTA PHYSIOLOGICA, 236, 931 - 933