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Cited by 32Virus-based gene therapy researchParkinson's Disease Mechanisms and TreatmentsRNA Interference and Gene Delivery

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

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Fajardo-Serrano, Ana, Rico, Alberto J., Roda, Elvira, Honrubia, Adriana, Arrieta, Sandra, Ariznabarreta, Goiaz, Chocarro, Julia, Lorenzo-Ramos, Elena +3 more·2021·International Journal of Molecular Sciences
DOI 10.3390/ijms22126389PMC PMC8232322
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Abstract

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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  • Serotype vectors with robust neuronal tropism and volumetric spread, such as AAV9, AAV8, and AAV1, are used for brain parenchyma administration and could aid with neurological illnesses that predominantly affect a specific brain region (e.g., Parkinson's disease).

    “Serotype vectors with robust neuronal tropism and volumetric spread, such as AAV9, AAV8, and AAV1, are used for this type of administration [80], and could certainly aid with neurological illnesses that predominantly affect a specific brain region (e.g.Parkinson's disease [81]).”

    From Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders (2022) · cites this paper as [81]

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Authors
Fajardo-Serrano, A., Rico, A. J., Roda, E., Honrubia, A., Arrieta, S., Ariznabarreta, G., Chocarro, J., Lorenzo-Ramos, E., Pejenaute, A., Vázquez, A., & Lanciego, J. L.
Affiliations
  • Centro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, Spain
  • Biomedical Research Networking Center on Neurodegenerative Diseases (ES)
  • Servicio Navarro de Salud (ES)
Corresponding author
Lanciego, José Luis
ORCID
Venue
International Journal of Molecular Sciences
Publisher
MDPI AG
Published
2021-06-15
Volume
22
Issue
12
Pages
6389
ISSN
1422-0067 (ISSN-L)
DOI
10.3390/ijms22126389
PMC
PMC8232322
OpenAlex
W3168460635
Cited by
32
References
178
Citations by year
2026: 5 · 2025: 10 · 2024: 4 · 2023: 6 · 2022: 5 · 2021: 2
FWCI
0.63
Type
journal article
Language
en
Open access
Licence
Locations
  • International Journal of Molecular Sciences (publishedVersion, cc-by)
  • PubMed Central (publishedVersion, cc-by)
  • PubMed (publishedVersion)
  • DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
Funders
  • European Research Council (ERC Adv 340527)
  • CiberNed's Intramural Program Grant (PI2017/02)
  • Ministerio de Ciencia, Innovación y Universidades (BFU2017/82407-R)
  • Departamento de Salud, Gobierno de Navarra (046-2017_NAB7, 011-1383-2019-000006)
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
  • Health Sciences › Medicine › Neurology › Parkinson's Disease Mechanisms and Treatments
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Keywords
adeno-associated virus, Parkinson's disease, AAV serotypes, synucleinopathies, gene therapy, disease modeling, neurodegenerative diseases, precision medicine
MeSH terms
Dependovirus, Animals, Disease Models, Animal, Genetic Vectors, Humans, Parkinson Disease, Gene Transfer Techniques, Biomedical Research

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