title: The translational potential of human induced pluripotent stem cells for clinical neurology : The translational potential of hiPSCs in neurology.
abstract: The induced pluripotent state represents a decade-old Nobel prize-winning discovery. Human-induced pluripotent stem cells (hiPSCs) are generated by the nuclear reprogramming of any somatic cell using a variety of established but evolving methods. This approach offers medical science unparalleled experimental opportunity to model an individual patient's disease "in a dish." HiPSCs permit developmentally rationalized directed differentiation into any cell type, which express donor cell mutation(s) at pathophysiological levels and thus hold considerable potential for disease modeling, drug discovery, and potentially cell-based therapies. This review will focus on the translational potential of hiPSCs in clinical neurology and the importance of integrating this approach with complementary model systems to increase the translational yield of preclinical testing for the benefit of patients. This strategy is particularly important given the expected increase in prevalence of neurodegenerative disease, which poses a major burden to global health over the coming decades.
authors.affiliation: Department of Molecular Neuroscience, UCL Institute of Neurology, Queen Square, London, WC1N3BG, UK.|Sobell Department of Motor Neuroscience and Movement Disorders, UCL Institute of Neurology, Queen Square, London, UK.
authors.forename: Helen
authors.initials: H
authors.lastname: Devine
authors.affiliation: Department of Molecular Neuroscience, UCL Institute of Neurology, Queen Square, London, WC1N3BG, UK. rickie.patani@ucl.ac.uk.|National Hospital for Neurology and Neurosurgery, UCL Institute of Neurology, Queen Square, London, WC1N 3BG, UK. rickie.patani@ucl.ac.uk.|Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK. rickie.patani@ucl.ac.uk.|Euan MacDonald Centre for MND, University of Edinburgh, Edinburgh, UK. rickie.patani@ucl.ac.uk.
authors.forename: Rickie
authors.initials: R
authors.lastname: Patani
mesh_terms: D002454:Cell Differentiation; D055808:Drug Discovery; D014644:Genetic Variation; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology*; D009462:Neurology; D057170:Translational Research, Biomedical
keywords: Cellular therapy; Disease modeling; Drug discovery; Human induced pluripotent stem cells (hiPSCs); Neurology; Translational medicine