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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| Format: | Article |
| Language: | English |
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MDPI AG
2021-06-01
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| Series: | International Journal of Molecular Sciences |
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| Online Access: | https://www.mdpi.com/1422-0067/22/12/6389 |
| _version_ | 1797530145471332352 |
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| author | Ana Fajardo-Serrano Alberto J. Rico Elvira Roda Adriana Honrubia Sandra Arrieta Goiaz Ariznabarreta Julia Chocarro Elena Lorenzo-Ramos Alvaro Pejenaute Alfonso Vázquez José Luis Lanciego |
| author_facet | Ana Fajardo-Serrano Alberto J. Rico Elvira Roda Adriana Honrubia Sandra Arrieta Goiaz Ariznabarreta Julia Chocarro Elena Lorenzo-Ramos Alvaro Pejenaute Alfonso Vázquez José Luis Lanciego |
| author_sort | Ana Fajardo-Serrano |
| collection | DOAJ |
| 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. |
| first_indexed | 2024-03-10T10:24:48Z |
| format | Article |
| id | doaj.art-c9b6623b8a9e4eeb9dc33097d3de3679 |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-10T10:24:48Z |
| publishDate | 2021-06-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-c9b6623b8a9e4eeb9dc33097d3de36792023-11-22T00:10:02ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672021-06-012212638910.3390/ijms22126389Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic UsesAna Fajardo-Serrano0Alberto J. Rico1Elvira Roda2Adriana Honrubia3Sandra Arrieta4Goiaz Ariznabarreta5Julia Chocarro6Elena Lorenzo-Ramos7Alvaro Pejenaute8Alfonso Vázquez9José Luis Lanciego10Centro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainCentro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainCentro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainCentro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainCentro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainCentro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainCentro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainCentro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainCentro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainInstituto de Investigación Sanitaria de Navarra (IdiSNA), 31008 Pamplona, SpainCentro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, SpainIt 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.https://www.mdpi.com/1422-0067/22/12/6389alpha-synucleinanimal modelneuroprotectiondisease-modifying therapyprecision medicine |
| spellingShingle | Ana Fajardo-Serrano Alberto J. Rico Elvira Roda Adriana Honrubia Sandra Arrieta Goiaz Ariznabarreta Julia Chocarro Elena Lorenzo-Ramos Alvaro Pejenaute Alfonso Vázquez José Luis Lanciego Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses International Journal of Molecular Sciences alpha-synuclein animal model neuroprotection disease-modifying therapy precision medicine |
| 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 | alpha-synuclein animal model neuroprotection disease-modifying therapy precision medicine |
| url | https://www.mdpi.com/1422-0067/22/12/6389 |
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