Stem Cells as In Vitro Model of Parkinson's Disease
Progress in understanding neurodegenerative cell biology in Parkinson's disease (PD) has been hampered by a lack of predictive and relevant cellular models. In addition, the lack of an adequate in vitro human neuron cell-based model has been an obstacle for the uncover of new drugs for treating...
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| Format: | Article |
| Language: | English |
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Wiley
2012-01-01
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| Series: | Stem Cells International |
| Online Access: | http://dx.doi.org/10.1155/2012/980941 |
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| _version_ | 1832558844513878016 |
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| author | Patricia L. Martínez-Morales Isabel Liste |
| author_facet | Patricia L. Martínez-Morales Isabel Liste |
| author_sort | Patricia L. Martínez-Morales |
| collection | DOAJ |
| description | Progress in understanding neurodegenerative cell biology in Parkinson's disease (PD) has been hampered by a lack of predictive and relevant cellular models. In addition, the lack of an adequate in vitro human neuron cell-based model has been an obstacle for the uncover of new drugs for treating PD. The ability to generate induced pluripotent stem cells (iPSCs) from PD patients and a refined capacity to differentiate these iPSCs into DA neurons, the relevant disease cell type, promises a new paradigm in drug development that positions human disease pathophysiology at the core of preclinical drug discovery. Disease models derived from iPSC that manifest cellular disease phenotypes have been established for several monogenic diseases, but iPSC can likewise be used for phenotype-based drug screens in complex diseases for which the underlying genetic mechanism is unknown. Here, we highlight recent advances as well as limitations in the use of iPSC technology for modelling PD “in a dish” and for testing compounds against human disease phenotypes in vitro. We discuss how iPSCs are being exploited to illuminate disease pathophysiology, identify novel drug targets, and enhance the probability of clinical success of new drugs. |
| format | Article |
| id | doaj-art-216c5d6d74b04bb8b5dba3e21d3a8855 |
| institution | Kabale University |
| issn | 1687-966X 1687-9678 |
| language | English |
| publishDate | 2012-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | Stem Cells International |
| spelling | doaj-art-216c5d6d74b04bb8b5dba3e21d3a88552025-02-03T01:31:25ZengWileyStem Cells International1687-966X1687-96782012-01-01201210.1155/2012/980941980941Stem Cells as In Vitro Model of Parkinson's DiseasePatricia L. Martínez-Morales0Isabel Liste1Unidad de Regeneración Neural, Área de Biología Celular y del Desarrollo, Centro Nacional de Microbiología, Instituto de Salud Carlos III (ISCIII), Majadahonda, 28220 Madrid, SpainUnidad de Regeneración Neural, Área de Biología Celular y del Desarrollo, Centro Nacional de Microbiología, Instituto de Salud Carlos III (ISCIII), Majadahonda, 28220 Madrid, SpainProgress in understanding neurodegenerative cell biology in Parkinson's disease (PD) has been hampered by a lack of predictive and relevant cellular models. In addition, the lack of an adequate in vitro human neuron cell-based model has been an obstacle for the uncover of new drugs for treating PD. The ability to generate induced pluripotent stem cells (iPSCs) from PD patients and a refined capacity to differentiate these iPSCs into DA neurons, the relevant disease cell type, promises a new paradigm in drug development that positions human disease pathophysiology at the core of preclinical drug discovery. Disease models derived from iPSC that manifest cellular disease phenotypes have been established for several monogenic diseases, but iPSC can likewise be used for phenotype-based drug screens in complex diseases for which the underlying genetic mechanism is unknown. Here, we highlight recent advances as well as limitations in the use of iPSC technology for modelling PD “in a dish” and for testing compounds against human disease phenotypes in vitro. We discuss how iPSCs are being exploited to illuminate disease pathophysiology, identify novel drug targets, and enhance the probability of clinical success of new drugs.http://dx.doi.org/10.1155/2012/980941 |
| spellingShingle | Patricia L. Martínez-Morales Isabel Liste Stem Cells as In Vitro Model of Parkinson's Disease Stem Cells International |
| title | Stem Cells as In Vitro Model of Parkinson's Disease |
| title_full | Stem Cells as In Vitro Model of Parkinson's Disease |
| title_fullStr | Stem Cells as In Vitro Model of Parkinson's Disease |
| title_full_unstemmed | Stem Cells as In Vitro Model of Parkinson's Disease |
| title_short | Stem Cells as In Vitro Model of Parkinson's Disease |
| title_sort | stem cells as in vitro model of parkinson s disease |
| url | http://dx.doi.org/10.1155/2012/980941 |
| work_keys_str_mv | AT patricialmartinezmorales stemcellsasinvitromodelofparkinsonsdisease AT isabelliste stemcellsasinvitromodelofparkinsonsdisease |