Cargando…

Gene Therapy: A Promising Approach for Neuroprotection in Parkinson’s Disease?

With the development of effective systems for gene delivery to the central nervous system (CNS), gene therapy has become a therapeutic option for the treatment of Parkinson’s disease (PD). Gene therapies that are the most advanced in the clinic have been designed to more effectively compensate for t...

Descripción completa

Detalles Bibliográficos
Autores principales: Valdés, Pamela, Schneider, Bernard L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5168434/
https://www.ncbi.nlm.nih.gov/pubmed/28066194
http://dx.doi.org/10.3389/fnana.2016.00123
_version_ 1782483342350876672
author Valdés, Pamela
Schneider, Bernard L.
author_facet Valdés, Pamela
Schneider, Bernard L.
author_sort Valdés, Pamela
collection PubMed
description With the development of effective systems for gene delivery to the central nervous system (CNS), gene therapy has become a therapeutic option for the treatment of Parkinson’s disease (PD). Gene therapies that are the most advanced in the clinic have been designed to more effectively compensate for the lack of dopamine signaling in the basal ganglia and rescue the cardinal motor symptoms of PD. However, it remains essential to devise novel therapies to prevent neurodegeneration and disease progression. Since gene therapy has been initially proposed for the delivery of neurotrophins to support the survival and function of dopaminergic neurons, our understanding of PD etiology has changed dramatically. Genes implicated in familial forms of the disease and genetic risk factors associated with sporadic PD have been identified. The spreading of the α-synuclein pathology, as well as perturbations of the lysosomal and mitochondrial activities, appear to play critical roles in the pathogenesis. These findings provide novel targets for gene therapy against PD, but at the same time underline the complexity of this chronic disease. Here we review and discuss the successes and limitations of gene therapy approaches, which have been proposed to provide neuroprotection in PD.
format Online
Article
Text
id pubmed-5168434
institution National Center for Biotechnology Information
language English
publishDate 2016
publisher Frontiers Media S.A.
record_format MEDLINE/PubMed
spelling pubmed-51684342017-01-06 Gene Therapy: A Promising Approach for Neuroprotection in Parkinson’s Disease? Valdés, Pamela Schneider, Bernard L. Front Neuroanat Neuroscience With the development of effective systems for gene delivery to the central nervous system (CNS), gene therapy has become a therapeutic option for the treatment of Parkinson’s disease (PD). Gene therapies that are the most advanced in the clinic have been designed to more effectively compensate for the lack of dopamine signaling in the basal ganglia and rescue the cardinal motor symptoms of PD. However, it remains essential to devise novel therapies to prevent neurodegeneration and disease progression. Since gene therapy has been initially proposed for the delivery of neurotrophins to support the survival and function of dopaminergic neurons, our understanding of PD etiology has changed dramatically. Genes implicated in familial forms of the disease and genetic risk factors associated with sporadic PD have been identified. The spreading of the α-synuclein pathology, as well as perturbations of the lysosomal and mitochondrial activities, appear to play critical roles in the pathogenesis. These findings provide novel targets for gene therapy against PD, but at the same time underline the complexity of this chronic disease. Here we review and discuss the successes and limitations of gene therapy approaches, which have been proposed to provide neuroprotection in PD. Frontiers Media S.A. 2016-12-20 /pmc/articles/PMC5168434/ /pubmed/28066194 http://dx.doi.org/10.3389/fnana.2016.00123 Text en Copyright © 2016 Valdés and Schneider. http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution and reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Neuroscience
Valdés, Pamela
Schneider, Bernard L.
Gene Therapy: A Promising Approach for Neuroprotection in Parkinson’s Disease?
title Gene Therapy: A Promising Approach for Neuroprotection in Parkinson’s Disease?
title_full Gene Therapy: A Promising Approach for Neuroprotection in Parkinson’s Disease?
title_fullStr Gene Therapy: A Promising Approach for Neuroprotection in Parkinson’s Disease?
title_full_unstemmed Gene Therapy: A Promising Approach for Neuroprotection in Parkinson’s Disease?
title_short Gene Therapy: A Promising Approach for Neuroprotection in Parkinson’s Disease?
title_sort gene therapy: a promising approach for neuroprotection in parkinson’s disease?
topic Neuroscience
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5168434/
https://www.ncbi.nlm.nih.gov/pubmed/28066194
http://dx.doi.org/10.3389/fnana.2016.00123
work_keys_str_mv AT valdespamela genetherapyapromisingapproachforneuroprotectioninparkinsonsdisease
AT schneiderbernardl genetherapyapromisingapproachforneuroprotectioninparkinsonsdisease