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Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses

It is without any doubt that precision medicine therapeutic strategies targeting neurodegenerative disorders are currently witnessing the spectacular rise of newly designed approaches based on the use of viral vectors as Trojan horses for the controlled release of a given genetic payload. Among the...

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Autores principales: Fajardo-Serrano, Ana, Rico, Alberto J., Roda, Elvira, Honrubia, Adriana, Arrieta, Sandra, Ariznabarreta, Goiaz, Chocarro, Julia, Lorenzo-Ramos, Elena, Pejenaute, Alvaro, Vázquez, Alfonso, Lanciego, José Luis
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8232322/
https://www.ncbi.nlm.nih.gov/pubmed/34203739
http://dx.doi.org/10.3390/ijms22126389
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author Fajardo-Serrano, Ana
Rico, Alberto J.
Roda, Elvira
Honrubia, Adriana
Arrieta, Sandra
Ariznabarreta, Goiaz
Chocarro, Julia
Lorenzo-Ramos, Elena
Pejenaute, Alvaro
Vázquez, Alfonso
Lanciego, José Luis
author_facet Fajardo-Serrano, Ana
Rico, Alberto J.
Roda, Elvira
Honrubia, Adriana
Arrieta, Sandra
Ariznabarreta, Goiaz
Chocarro, Julia
Lorenzo-Ramos, Elena
Pejenaute, Alvaro
Vázquez, Alfonso
Lanciego, José Luis
author_sort Fajardo-Serrano, Ana
collection PubMed
description It is without any doubt that precision medicine therapeutic strategies targeting neurodegenerative disorders are currently witnessing the spectacular rise of newly designed approaches based on the use of viral vectors as Trojan horses for the controlled release of a given genetic payload. Among the different types of viral vectors, adeno-associated viruses (AAVs) rank as the ones most commonly used for the purposes of either disease modeling or for therapeutic strategies. Here, we reviewed the current literature dealing with the use of AAVs within the field of Parkinson’s disease with the aim to provide neuroscientists with the advice and background required when facing a choice on which AAV might be best suited for addressing a given experimental challenge. Accordingly, here we will be summarizing some insights on different AAV serotypes, and which would be the most appropriate AAV delivery route. Next, the use of AAVs for modeling synucleinopathies is highlighted, providing potential readers with a landscape view of ongoing pre-clinical and clinical initiatives pushing forward AAV-based therapeutic approaches for Parkinson’s disease and related synucleinopathies.
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spelling pubmed-82323222021-06-26 Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses Fajardo-Serrano, Ana Rico, Alberto J. Roda, Elvira Honrubia, Adriana Arrieta, Sandra Ariznabarreta, Goiaz Chocarro, Julia Lorenzo-Ramos, Elena Pejenaute, Alvaro Vázquez, Alfonso Lanciego, José Luis Int J Mol Sci Review It is without any doubt that precision medicine therapeutic strategies targeting neurodegenerative disorders are currently witnessing the spectacular rise of newly designed approaches based on the use of viral vectors as Trojan horses for the controlled release of a given genetic payload. Among the different types of viral vectors, adeno-associated viruses (AAVs) rank as the ones most commonly used for the purposes of either disease modeling or for therapeutic strategies. Here, we reviewed the current literature dealing with the use of AAVs within the field of Parkinson’s disease with the aim to provide neuroscientists with the advice and background required when facing a choice on which AAV might be best suited for addressing a given experimental challenge. Accordingly, here we will be summarizing some insights on different AAV serotypes, and which would be the most appropriate AAV delivery route. Next, the use of AAVs for modeling synucleinopathies is highlighted, providing potential readers with a landscape view of ongoing pre-clinical and clinical initiatives pushing forward AAV-based therapeutic approaches for Parkinson’s disease and related synucleinopathies. MDPI 2021-06-15 /pmc/articles/PMC8232322/ /pubmed/34203739 http://dx.doi.org/10.3390/ijms22126389 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Fajardo-Serrano, Ana
Rico, Alberto J.
Roda, Elvira
Honrubia, Adriana
Arrieta, Sandra
Ariznabarreta, Goiaz
Chocarro, Julia
Lorenzo-Ramos, Elena
Pejenaute, Alvaro
Vázquez, Alfonso
Lanciego, José Luis
Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses
title Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses
title_full Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses
title_fullStr Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses
title_full_unstemmed Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses
title_short Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses
title_sort adeno-associated viral vectors as versatile tools for parkinson’s research, both for disease modeling purposes and for therapeutic uses
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8232322/
https://www.ncbi.nlm.nih.gov/pubmed/34203739
http://dx.doi.org/10.3390/ijms22126389
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