Exp Neurol. 2013 Jan 5. pii: S0014-4886(13)00004-6. doi: 10.1016/j.expneurol.2012.12.017. [Epub ahead of print]
Ali F, Stott SR, Barker RA.
Source
Cambridge Centre for Brain Repair, E.D. Adrian Building, Forvie Site, Robinson Way, Cambridge CB2 0PY, England.
Abstract
Progress in Parkinson's disease (PD) research has been hampered by the lack of an appropriate model which exhibits the core pathology seen in the human brain. Recent advances in deriving cells with neuronal phenotypes from patients with neurodegenerative disorders through cellular reprogramming offers a unique tool for disease modelling and may help shed light on the molecular pathogenesis the drives the progression of the disease. This technology may also help in establishing platforms for drug screening and open up exciting new prospects for cell grafting. In this review, we will discuss progress made in differentiating stem cells into authentic dopamine neurons and where we stand with respect to clinical trials with these cells in patients with PD. We will also examine the various approaches used in cellular reprogramming and their differentiation into patient-specific midbrain dopamine neurons, with an emphasis particularly on modelling familial cases of PD to recapitulate disease phenotypes. This review will highlight some of the challenges that need to be addressed for this technology to have any potential clinical application in cell therapy and personalised medicine.
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