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
Defective platelet function in Niemann-Pick disease type C1
Chen OCW. et al, (2020), JIMD Reports, 56, 46 - 57
Upregulating β-hexosaminidase activity in rodents prevents α-synuclein lipid associations and protects dopaminergic neurons from α-synuclein-mediated neurotoxicity.
PLATT F. and PRIESTMAN D., (2020), Acta Neuropathologica Communications
Acetyl-Leucine slows disease progression in lysosomal storage disorders
PLATT F. et al, (2020), Brain Communications
Unbiased yeast screens identify cellular pathways affected in Niemann-Pick disease type C.
Colaco A. et al, (2020), Life Sci Alliance, 3
Defective Platelet Function in Niemann-Pick disease type C1
COLACO A. and PLATT F., (2020), JIMD Communications
Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease.
Colaco A. et al, (2020), J Inherit Metab Dis, 43, 574 - 585
Beneficial Effects of Acetyl-DL-Leucine (ADLL) in a Mouse Model of Sandhoff Disease
PLATT F. et al, (2020), Journal of Clinical Medicine