Principal Investigators

    Institution

    Contact information of lead PI

    Country

    European Commission

    Title of project or programme

    New experimental therapeutic approaches for Parkinson’s disease by direct DA neuronal reprogramming

    Source of funding information

    European Commission FP7-Seventh Framework Programme

    Total sum awarded (Euro)

    € 2,415,767

    Start date of award

    01/06/2014

    Total duration of award in years

    5.0

    The project/programme is most relevant to:

    Parkinson's disease & PD-related disorders

    Keywords

    Research Abstract

    Neurodegenerative diseases cause a significant burden on the elderly population in Europe. Parkinson’s disease (PD) affects 1.2 million people in Europe and with the increasing life expectancy this number will rise, putting more pressure on health care. Treatment of PD is only symptomatic, and therefore, there is an urgent need for more efficient therapies. Degeneration of mesencephalic DA neurons triggers the initial phases of PD, which raises the concept that cell replacement might represent a long-term restorative option for this neuropathology. Indeed, previous studies in PD patients have indicated that cell therapy has the potential to significantly sustain an enduring symptomatic relief. However, these studies suffered for lacking an ideal source of transplantable human DA neurons. Only recently the generation of induced stem cells (iPSCs) by the reprogramming of somatic cells has disclosed the possibility to generate individual specific neurons with a high therapeutic potential. We have recently developed a methodology that promotes transdifferentiation of mouse and human fibroblasts into functional induced dopaminergic neuronal (iDAN) cells. iDAN cells display sophisticated neuronal properties including pacemaking firing activity, synaptic integration, activity-dependent dopamine release and D2 functional autoreceptors. Therefore, iDAN cells offer an unprecedented cellular source with ideal features for cell therapy in PD, since they can be generated from the patients in high amounts. Here, we propose to strengthen the technology in the human setting and decipher the molecular events. Next, we will elaborate methods of in vivo reprogramming promoting neuronal transdifferentiation locally in the mouse brain. Finally, autologous transplantations of iDAN cells in parkinsonian monkeys will be attempted. Overall, this project will enhance cell reprogramming technologies with the ambition to generate a superior cellular source for transplantations in PD patients.

    Lay Summary

    Further information available at:

Types: Investments > €500k
Member States: European Commission
Diseases: Parkinson's disease & PD-related disorders
Years: 2016
Database Categories: N/A
Database Tags: N/A

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