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

Types: Investments > €500k
Member States: United Kingdom
Diseases: Motor neurone diseases
Years: 2011
Database Categories: N/A
Database Tags: N/A

Export as PDF