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...

Full description

Bibliographic Details
Main Authors: Pignataro, D. (Diego), González-Aseguinolaza, G. (Gloria), Lanciego, J.L. (José Luis)
Format: info:eu-repo/semantics/doctoralThesis
Language:eng
Published: 2018
Subjects:
Online Access:https://hdl.handle.net/10171/50966
_version_ 1793400460944605184
author Pignataro, D. (Diego)
González-Aseguinolaza, G. (Gloria)
Lanciego, J.L. (José Luis)
author_facet Pignataro, D. (Diego)
González-Aseguinolaza, G. (Gloria)
Lanciego, J.L. (José Luis)
author_sort Pignataro, D. (Diego)
collection DSpace
description 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.
format info:eu-repo/semantics/doctoralThesis
id oai:dadun.unav.edu:10171-50966
institution Universidad de Navarra
language eng
publishDate 2018
record_format dspace
spelling oai:dadun.unav.edu:10171-509662021-07-09T07:09:13Z rAAV8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases Pignataro, D. (Diego) González-Aseguinolaza, G. (Gloria) Lanciego, J.L. (José Luis) neurodegenerative diseases Parkinson gene therapy Materias Investigacion::Ciencias de la Salud::Enfermedades y ataques cerbrovasculares 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. 2018-04-11T11:31:19Z 2018-04-11T11:31:19Z 2018-04-11 2017-04-10 info:eu-repo/semantics/doctoralThesis https://hdl.handle.net/10171/50966 eng info:eu-repo/semantics/openAccess application/pdf
spellingShingle neurodegenerative diseases
Parkinson
gene therapy
Materias Investigacion::Ciencias de la Salud::Enfermedades y ataques cerbrovasculares
Pignataro, D. (Diego)
González-Aseguinolaza, G. (Gloria)
Lanciego, J.L. (José Luis)
rAAV8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases
title rAAV8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases
title_full rAAV8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases
title_fullStr rAAV8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases
title_full_unstemmed rAAV8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases
title_short rAAV8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases
title_sort raav8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases
topic neurodegenerative diseases
Parkinson
gene therapy
Materias Investigacion::Ciencias de la Salud::Enfermedades y ataques cerbrovasculares
url https://hdl.handle.net/10171/50966
work_keys_str_mv AT pignataroddiego raav8mediatedinvivoreprogrammingofstriatalastrocytesintoneuronsasapotentialtherapeuticstrategyforneurodegenerativediseases
AT gonzalezaseguinolazaggloria raav8mediatedinvivoreprogrammingofstriatalastrocytesintoneuronsasapotentialtherapeuticstrategyforneurodegenerativediseases
AT lanciegojljoseluis raav8mediatedinvivoreprogrammingofstriatalastrocytesintoneuronsasapotentialtherapeuticstrategyforneurodegenerativediseases