Creating a new model of MND using stem cell technology

Title of project or programme

Title of PI

Creating a new model of MND using stem cell technology

Principal Investigators of project/programme grant

Title	Forname	Surname	Institution	Country
Professor	Siddarthan	Chandran	University of Edinburgh	UK
Professor	Ian	Wilmut	University of Edinburgh	UK
Professor	Tom	Maniatis	Columbia University	USA
Professor	Christopher	Shaw	King’s College London	UK

Address of institution of lead PI

Institution	University of Edinburgh, Chancellors Building
Street Address	47 Little France Crescent
City	Edinburgh
Postcode	EH16 4SB

Country

• United Kingdom

Total sum awarded (Euro)
960000
Start date of award
01-06-2010
Total duration of award in months
36
The project/programme is most relevant to

• Motor neurone diseases

Keywords
fibroblasts, induced pluripotential stem cells, iPS cells, motor neurons, astrocytes
Research abstract in English
The discovery that TDP-43 is the major protein within ubiquitinated cytoplasmic inclusions – the hallmark pathology in 90% of familial and sporadic cases – and the recent identification of causitive TDP-43 mutations in 1-4% of familial and sporadic cases, highlights the need for cellular models to address the consequences of mutant TDP-43 in both motor neurons and astrocytes. Recent discoveries in reprogramming and resulting derivation of human iPS cell lines permit human-based models to study the consequences of disease-causing mutations. Against this background, our derivation and validation of TDP-43 mutant and control iPS lines offers new opportunities ofr the development of an in vitro human disease model of MND. specifically, this collaborative project will exploit the unique opportunity of deriving patient-specific motor neurons and astrocytes carrying disease causing TDP-43 mutations in order to addess key questions concerning autonomous and non-cell autonomous mechanisms of motor neuron degeneration.
Lay summary

In which category does this research fall?

• Basic research

Creating a new model of MND using stem cell technology

Investments > €500k
United Kingdom
Motor neurone diseases
2011