rAAV8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases
CNS neurodegenerative disorders represent a major health problem worldwide. In PD, the progressive loss of miDA neurons leads to an irreversible dopamine deficiency in the striatum, ultimately triggering the appearance of the cardinal motor symptoms that typically characterize this movement disorder...
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| Tipo de recurso: | tesis doctoral |
| Fecha de publicación: | 2017 |
| País: | España |
| Institución: | Universidad de Navarra |
| Repositorio: | Dadun. Depósito Académico Digital de la Universidad de Navarra |
| Idioma: | inglés |
| OAI Identifier: | oai:dadun.unav.edu:10171/50966 |
| Acceso en línea: | https://hdl.handle.net/10171/50966 |
| Access Level: | acceso abierto |
| Palabra clave: | neurodegenerative diseases Parkinson gene therapy Materias Investigacion::Ciencias de la Salud::Enfermedades y ataques cerbrovasculares |
| Sumario: | CNS neurodegenerative disorders represent a major health problem worldwide. In PD, the progressive loss of miDA neurons leads to an irreversible dopamine deficiency in the striatum, ultimately triggering the appearance of the cardinal motor symptoms that typically characterize this movement disorder of basal ganglia origin. Accordingly, the so-called in vivo direct reprogramming has recently emerged as an appealing technical choice to further generate functional DA neurons from endogenous glial cells in situ. In this regard, here we are taking advantage of newly-designed adeno-associated viral vectors in an attempt to overexpress a number of transcription factors within striatal astrocytes to compensate the lack of dopamine in this brain region. The main goal of this project is the development of new gene therapy tools to drive gene expression in specific cell populations of the CNS for future applications in neurodegenerative diseases including Parkinson. |
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