Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9
Abstract
Several attempts have been made to discover the ideal vector for gene therapy in central nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle since they exhibit stable transgene expression in post-mitotic cells, neuronal tropism, low risk of insertional mutagenesis and diminished immune responses. Additionally, the discovery that a particular serotype, AAV9, bypasses the blood-brain barrier has raised the possibility of intravascular administration as a non-invasive delivery route to achieve widespread CNS gene expression. AAV9 intravenous delivery has already shown promising results for several diseases in animal models, including lysosomal storage disorders and motor neuron diseases, opening the way to the first clinical trial in the field. This review presents an overview of clinical trials for CNS disorders using AAVs and will focus on preclinical studies based on the systemic gene delivery using AAV9.
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IV delivery is a non-invasive method and could take advantage of the dense vascular network in the brain and spinal cord for more uniform vector distribution but would require vectors to circumvent the obstacle of the BBB.
“IV delivery is a non-invasive method and could take advantage of the dense vascular network in the brain and spinal cord for more uniform vector distribution but would require vectors to circumvent the obstacle of BBB [110].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [110]
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- Authors
- Saraiva, J., Nobre, R. J., & Pereira de Almeida, L.
- Affiliations
- Centro de Neurociências e Biologia Celular (PT)
- Venue
- Journal of Controlled Release
- Publisher
- Elsevier BV
- Published
- 2016-11
- Volume
- 241
- Pages
- 94-109
- ISSN
- 0168-3659 (ISSN-L)
- OpenAlex
- W2519081550
- Cited by
- 201
- References
- 134
- Citations by year
- 2026: 6 · 2025: 22 · 2024: 21 · 2023: 23 · 2022: 18 · 2021: 22 · 2020: 23 · 2019: 28 · 2018: 24 · 2017: 12 · 2016: 1
- FWCI
- 2.92
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Journal of Controlled Release (publishedVersion)
- PubMed (publishedVersion)
- Funders
- FEDER
- Competitive Factors Operational Program – COMPETE
- Portuguese Foundation for Science and Technology (PTDC/SAU-NMC/116512/2010, E-Rare4/0003/2012, PTDC/NEU-NMC/0084/2014, EXPL/NEU-NMC/0331/2012, SFRH/BPD/66705/2009)
- JPND (JPCOFUND/0001, 0005/2015)
- Richard Chin and Lily Lock Machado Joseph Disease Research Fund
- National Ataxia Foundation
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Keywords
- gene therapy, central nervous system, AAV9, adeno-associated virus vectors, systemic drug delivery, intravenous administration, blood-brain barrier bypass, lysosomal storage disease, motor neuron disease, clinical trials, preclinical research
- MeSH terms
- Dependovirus, Animals, Central Nervous System Diseases, Clinical Trials as Topic, Genetic Vectors, Humans, Genetic Therapy, Gene Transfer Techniques, Capsid Proteins
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