Name of Fellow


CATARINA SOFIA OLIVEIRA MIRANDA

Institution


Funder


FCT

Contact information of fellow


Country


Portugal

Title of project/programme


MESENCHYMAL STEM CELLS FOR MACHADO-JOSEPH DISEASE THERAPY

Source of funding information


FCT

Total sum awarded (Euro)


€ 116,640

Start date of award


01/01/13

Total duration of award in years


6.0

The project/programme is most relevant to:


Spinocerebellar ataxia (SCA)

Keywords


Research Abstract


Mesenchymal stromal cells (MSCs) are an extremely promising tool for therapy of neurodegenerative disorders. However, this therapeutic approach has received limited attention with respect to spinocerebellar ataxias (SCAs). In the present study, we aim at investigating the potential of MSC systemic transplantation as a cellular therapy for Machado-Joseph disease/spinocerebellar ataxia type 3 (MJD/SCA-3), the most prevalent genetically-inherited SCA worldwide. For this purpose, we will evaluate the engraftment and neuroprotective/neuroregenerative effects of wild-type MSC transplantation both in in vitro and in vivo models of MJD and further dissect its protective mechanisms. In addition, we will evaluate if the neuroprotective potential of MJD MSCs is impaired and whether gene correction produce enhanced positive outcomes. Moreover, treatment with granulocyte colony-stimulating factor (G-CSF) will be compared with MSC transplantation. We expect to provide evidence that BM-MSCs can alleviate MJD and become a good candidate for disease-modifying MJD therapies, so far inexistent.

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