Patient-specific treatment for Parkinson's disease using reprogrammed skin cells
Swedish Research Council
Parkinson's disease & PD-related disorders
We have developed a new reprogramming technique that bypass the pluripotent stage and directly converts fibroblasts into dopamine neurons. Our findings open up for possibilities to generate patient specific neurons on demand. In this proposal, we will build on our recent achievements, with the aim to develop direct neural conversion for use in cell therapy for Parkinson´s disease. Our team consists of basic scientists, medical ethicists, neurologists and neurosurgeons all with documented expertise in translational research and cell replacement therapy in PD. Working together, our team will guide the basic science and pre-clinical evaluation work such that issues critical for clinical application of directly reprogrammed neurons are adequately and specifically addressed. Additionally, we will define and analyze new ethical and societal issues related to the use of directly converted fibroblasts as a source of neurons for brain repair in PD and provide a road map for translating directly programmed DA neurons for use in patients with PD in the clinic. As cellular reprogramming becomes more finely controlled, efficient and safe, it seems inevitable that patients will some day be treated with healthy versions of their own cells. The outcome of this project will aid this process both by developing the technique so that it is efficient, safe and compatible with clinical use and by developing the ethical and clinical framework and guidelines required for such clinical applications.