title: Induced pluripotent stem cell-based studies of Parkinson's disease: challenges and promises.
abstract: A critical step in the development of effective therapeutics to treat Parkinson's disease (PD) is the identification of molecular pathogenic mechanisms underlying this chronically progressive neurodegenerative disease. However, while animal models have provided valuable information about the molecular basis of PD, the lack of faithful cellular and animal models that recapitulate human pathophysiology is delaying the development of new therapeutics. The reprogramming of somatic cells to induced pluripotent stem cells (iPSC) using delivery of defined combinations of transcription factors is a groundbreaking discovery that opens great opportunities for modeling human diseases, including PD, since iPSC can be generated from patients and differentiated into disease-relevant cell types, which would capture the patients' genetic complexity. Furthermore, human iPSC-derived neuronal models offer unprecedented access to early stages of the disease, allowing the investigation of the events that initiate the pathologic process in PD. Recently, human iPSC-derived neurons from patients with familial and sporadic PD have been generated and importantly they recapitulate some PD-related cell phenotypes, including abnormal α-synuclein accumulation in vitro, and alterations in the autophagy machinery. This review highlights the current PD iPSC-based models and discusses the potential future research directions of this field.
authors.forename: Adriana
authors.initials: A
authors.lastname: Sanchez-Danes
authors.forename: Patrizia
authors.initials: P
authors.lastname: Benzoni
authors.forename: Maurizio
authors.initials: M
authors.lastname: Memo
authors.forename: Patrizia
authors.initials: P
authors.lastname: Dell'Era
authors.forename: Angel
authors.initials: A
authors.lastname: Raya
authors.affiliation: Institute for Biomedicine of the University of Barcelona (IBUB), Barcelona Science Park, Baldiri Reixac 15-21, 08028 Barcelona, Spain. aconsiglio@pcb.ub.es.
authors.forename: Antonella
authors.initials: A
authors.lastname: Consiglio
mesh_terms: D000818:Animals; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000276:immunology / Q000637:transplantation*; D058953:Neural Stem Cells / Q000276:immunology / Q000473:pathology / Q000637:transplantation*; D010300:Parkinson Disease / Q000175:diagnosis / Q000276:immunology / Q000473:pathology* / Q000628:therapy*; D039904:Pluripotent Stem Cells / Q000276:immunology / Q000473:pathology / Q000637:transplantation; D033581:Stem Cell Transplantation / Q000379:methods*