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An induced pluripotent stem cell-based neuronal model of Spinocerebellar ataxia

Total Cost €


EC-Contrib. €






 iPSCAtaxia project word cloud

Explore the words cloud of the iPSCAtaxia project. It provides you a very rough idea of what is the project "iPSCAtaxia" about.

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Project "iPSCAtaxia" data sheet

The following table provides information about the project.


Organization address
city: OXFORD
postcode: OX1 2JD

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country United Kingdom [UK]
 Project website
 Total cost 195˙454 €
 EC max contribution 195˙454 € (100%)
 Programme 1. H2020-EU.1.3.2. (Nurturing excellence by means of cross-border and cross-sector mobility)
 Code Call H2020-MSCA-IF-2015
 Funding Scheme MSCA-IF-EF-ST
 Starting year 2016
 Duration (year-month-day) from 2016-07-01   to  2019-01-23


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 


 Project objective

The spinocerebellar ataxias (SCAs) are a diverse group of neurological disorders defined by a loss of motor coordination. No effective treatments exist, and there is thus a pressing need for suitable models in which to study disease progression and to screen potential therapies. Induced pluripotent stem cells (iPSCs), which are capable of differentiation into any cell type of the body, offer the opportunity to study neurodegeneration in vitro. The proposed study aims to be the first in Europe to establish an iPSC-derived model of cerebellar disease, focusing on two genetic subtypes of SCA - SCA14 and 41. Increasing evidence points towards common pathological pathways shared across multiple SCA subtypes, which could provide novel therapeutic targets. There is also evidence to suggest that abnormalities in Purkinje cell development may contribute to the pathogenesis of the SCAs. This model will thus be used to elucidate the molecular mechanisms underlying common SCA-causing disease pathways, and to unravel the neurodevelopmental aspects of these diseases, in order to develop early-intervention therapies. Preliminary results indicate successful differentiation of iPSCs into cerebellar progenitor cells, using a protocol I have optimised. Following terminal differentiation, these cells will be investigated for common disease phenotypes and developmental defects, using a combination of transcriptomic, biochemical, and electrophysiological methods. Results will be validated by comparison with post-mortem brain samples and ataxic mouse models. By adapting this differentiation strategy to a format amenable to high-throughput screening, a pipeline will be established for the identification of novel therapeutic compounds. This groundbreaking study will enable the investigation of mechanisms which render the cells of the cerebellum particularly sensitive to degeneration, as well as providing a platform for pre-clinical screening of therapeutics for neurodegenerative conditions.


year authors and title journal last update
List of publications.
2018 Maggie M. K. Wong, Stephanie D. Hoekstra, Jane Vowles, Lauren M. Watson, Geraint Fuller, Andrea H. Németh, Sally A. Cowley, Olaf Ansorge, Kevin Talbot, Esther B. E. Becker
Neurodegeneration in SCA14 is associated with increased PKCγ kinase activity, mislocalization and aggregation
published pages: , ISSN: 2051-5960, DOI: 10.1186/s40478-018-0600-7
Acta Neuropathologica Communications 6/1 2019-09-27
2018 Lauren M. Watson, Maggie M. K. Wong, Jane Vowles, Sally A. Cowley, Esther B. E. Becker
A Simplified Method for Generating Purkinje Cells from Human-Induced Pluripotent Stem Cells
published pages: 419-427, ISSN: 1473-4222, DOI: 10.1007/s12311-017-0913-2
The Cerebellum 17/4 2019-09-27
2017 Lauren M. Watson, Elizabeth Bamber, Ricardo Parolin Schnekenberg, Jonathan Williams, Conceição Bettencourt, Jennifer Lickiss, Sandeep Jayawant, Katherine Fawcett, Samuel Clokie, Yvonne Wallis, Penny Clouston, David Sims, Henry Houlden, Esther B.E. Becker, Andrea H. Németh
Dominant Mutations in GRM1 Cause Spinocerebellar Ataxia Type 44
published pages: 451-458, ISSN: 0002-9297, DOI: 10.1016/j.ajhg.2017.08.005
The American Journal of Human Genetics 101/3 2019-09-27
2017 Esther Becker
Recent advances in modelling of cerebellar ataxia using induced pluripotent stem cells
published pages: 11-15, ISSN: 2572-942X, DOI: 10.29245/2572.942X/2017/7.1134
Journal of Neurology and Neuromedicine 2/7 2019-09-27

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