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.
Keywords: Induced pluripotent stem cells, Parkinson’s disease, neurodegenerative disease, human cellular model.
CNS & Neurological Disorders - Drug Targets
Title:Induced Pluripotent Stem Cell-Based Studies of Parkinson's Disease: Challenges and Promises
Volume: 12 Issue: 8
Author(s): Adriana Sanchez-Danes, Patrizia Benzoni, Maurizio Memo, Patrizia Dell’Era, Angel Raya and Antonella Consiglio
Affiliation:
Keywords: Induced pluripotent stem cells, Parkinson’s disease, neurodegenerative disease, human cellular model.
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.
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Cite this article as:
Sanchez-Danes Adriana, Benzoni Patrizia, Memo Maurizio, Dell’Era Patrizia, Raya Angel and Consiglio Antonella, Induced Pluripotent Stem Cell-Based Studies of Parkinson's Disease: Challenges and Promises, CNS & Neurological Disorders - Drug Targets 2013; 12 (8) . https://dx.doi.org/10.2174/187152731131200128
DOI https://dx.doi.org/10.2174/187152731131200128 |
Print ISSN 1871-5273 |
Publisher Name Bentham Science Publisher |
Online ISSN 1996-3181 |
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