Research groups
Colleges
Frances Platt
FRS FMedSci
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.
She was elected a fellow of the Academy of Medical Sciences in 2011 and was the recipient of a Royal Society Wolfson Merit Award in 2013. In 2016 she became a Wellcome Trust Investigator in Science. She was appointed Head of the Department of Pharmacology in 2020 and was elected a Fellow of the Royal Society in 2021. She was awarded the Thudichum Medal in 2023 by the Biochemical Society, UK.
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
Recent publications
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Deletion of GBA in neurons, but not microglia, causes neurodegeneration in a Gaucher mouse model.
Journal article
Duffy HBD. et al, (2024), JCI Insight
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The annotation of GBA1 has been concealed by its protein-coding pseudogene GBAP1.
Journal article
Gustavsson EK. et al, (2024), Sci Adv, 10
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Compartmentalisation proteomics revealed endolysosomal protein network changes in a goat model of atrial fibrillation
Journal article
PLATT F., (2024), iScience
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Plasma phosphorylated-tau217 is increased in Niemann-Pick disease type C.
Journal article
Gonzalez-Ortiz F. et al, (2024), Brain Commun, 6
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Cholera intoxication of human enteroids reveals interplay between decoy and functional glycoconjugate ligands.
Journal article
Singla A. et al, (2023), Glycobiology, 33, 801 - 816
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The expanding boundaries of sphingolipid lysosomal storage diseases; insights from Niemann-Pick disease type C.
Journal article
Platt FM., (2023), Biochem Soc Trans
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NPC1 plays a role in the trafficking of specific cargo to melanosomes.
Journal article
Rus AA. et al, (2023), J Biol Chem, 299