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
Molecular and biochemical insights into dysregulation of glycosphingolipid metabolism in a mouse model of lysosomal free sialic acid storage disorder.
Journal article
Sabir MS. et al, (2026), Exp Neurol, 399
Human iPSC Models of Ganglioside Deficiency Reveal a Sialylated Lipid Requirement for Plasma-Membrane Organization and Neuronal Activity
Preprint
Barrow HG. et al, (2026)
Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration.
Journal article
Liu W. et al, (2026), Hum Gene Ther
Miglustat as a Treatment for Adults with Tangier Disease Neuropathy: The MUSTANG N-of-1 Trial with 21 months Clinical Observation.
Journal article
Cook A. et al, (2026), Neurol Ther, 15, 447 - 456
Links between COVID-19, long COVID, and neurodegeneration: The role of glycosphingolipids.
Journal article
Spedding M. et al, (2026), Pharmacol Rev, 78
Limited therapeutic efficacy of N-acetyl-L-leucine in a mouse model of CLN1 disease.
Journal article
Ziółkowska EA. et al, (2025), Sci Rep, 16
N-acetyl-L-leucine (Levacetylleucine) normalizes Transcription Factor EB (TFEB) activity by stereospecific bidirectional modulation
Preprint
Davis LC. et al, (2025)
