Title of project or programme
Title of PICreating a new model of MND using stem cell technology
Principal Investigators of project/programme grant
ProfessorSiddarthanChandranUniversity of EdinburghUK
ProfessorIanWilmutUniversity of EdinburghUK
ProfessorTomManiatisColumbia UniversityUSA
ProfessorChristopherShawKing’s College LondonUK
Address of institution of lead PI
InstitutionUniversity of Edinburgh, Chancellors Building
Street Address47 Little France Crescent
PostcodeEH16 4SB
  • United Kingdom
Total sum awarded (Euro)


Start date of award


Total duration of award in months


The project/programme is most relevant to
  • Motor neurone diseases

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

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