F
Frances M. Platt
Researcher at University of Oxford
Publications - 320
Citations - 23719
Frances M. Platt is an academic researcher from University of Oxford. The author has contributed to research in topics: Lysosomal storage disease & Substrate reduction therapy. The author has an hindex of 78, co-authored 298 publications receiving 21542 citations. Previous affiliations of Frances M. Platt include Washington University in St. Louis & Thomas Jefferson University.
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Journal ArticleDOI
bcl-2-immunoglobulin transgenic mice demonstrate extended B cell survival and follicular lymphoproliferation.
Timothy J. McDonnell,Natasha Deane,Frances M. Platt,Gabriel Núñez,Ulrich Jaeger,John P. McKearn,Stanley J. Korsmeyer +6 more
TL;DR: In this article, minigene constructs representing the bcl-2-Ig fusion gene found at this chromosomal breakpoint were placed into the germ line of mice to assess the effects of the t(14;18) interchromosomal translocation during development.
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Autophagy Induction and Autophagosome Clearance in Neurons: Relationship to Autophagic Pathology in Alzheimer's Disease
Barry Boland,Ashok Kumar,Sooyeon Lee,Frances M. Platt,Jerzy Wegiel,Wai Haung Yu,Ralph A. Nixon +6 more
TL;DR: It is shown that constitutive macroautophagy in primary cortical neurons is highly efficient, because newly formed autophagosomes are rapidly cleared by fusion with lysosomes, accounting for their scarcity in the healthy brain, and that the autophagic pathology observed in AD most likely arises from impaired clearance of AVs rather than strong autophagy induction alone.
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Lysosomal storage diseases
TL;DR: Lysosomal storage diseases (LSDs) are a group of over 70 diseases characterized by lysosome dysfunction, most of which are inherited as autosomal recessive traits.
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Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium
Emyr Lloyd-Evans,Anthony J. Morgan,Xingxuan He,David Smith,Elena Elliot-Smith,Daniel J. Sillence,Grant C. Churchill,Edward H. Schuchman,Antony Galione,Frances M. Platt +9 more
TL;DR: It is found that NPC1-mutant cells have a large reduction in the acidic compartment calcium store compared to wild-type cells, which represents a new target for therapeutic intervention, as elevation of cytosolic calcium with curcumin normalized NPC1 disease cellular phenotypes and prolonged survival of the NPC1 mouse.
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Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis
Timothy M. Cox,Robin H. Lachmann,C. E. M. Hollak,Johannes M. F. G. Aerts,S. van Weely,M. Hrebicek,Frances M. Platt,Terry D. Butters,Raymond A. Dwek,C. Moyses,I. Gow,Deborah Elstein,Ari Zimran +12 more
TL;DR: Reducing the rate of substrate formation by OGT 918 improves key clinical features of non-neuronopathic Gaucher's disease and justifies further trials in this and other glycosphingolipid storage disorders.