Research groups
Colleges
Frances Platt
Ph.D
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
Recent publications
-
Defective platelet function in Niemann-Pick disease type C1
Journal article
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.
Journal article
PLATT F. and PRIESTMAN D., (2020), Acta Neuropathologica Communications
-
Acetyl-Leucine slows disease progression in lysosomal storage disorders
Journal article
PLATT F. et al, (2020), Brain Communications
-
Unbiased yeast screens identify cellular pathways affected in Niemann-Pick disease type C.
Journal article
Colaco A. et al, (2020), Life Sci Alliance, 3
-
Defective Platelet Function in Niemann-Pick disease type C1
Journal article
COLACO A. and PLATT F., (2020), JIMD Communications
-
Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease.
Journal article
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
Journal article
PLATT F. et al, (2020), Journal of Clinical Medicine