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Clinical Applications Involving CNS Gene Transfer
Open accessAbstract
Diseases of the central nervous system (CNS) have traditionally been the most difficult to treat by traditional pharmacological methods, due mostly to the blood-brain barrier and the difficulties associated with repeated drug administration targeting the CNS. Viral vector gene transfer represents a way to permanently provide a therapeutic protein within the nervous system after a single administration, whether this be a gene replacement strategy for an inherited disorder or a disease-modifying protein for a disease such as Parkinson's. Gene therapy approaches for CNS disorders has evolved considerably over the last two decades. Although a breakthrough treatment has remained elusive, current strategies are now considerably safer and potentially much more effective. This chapter will explore the past, current, and future status of CNS gene therapy, focusing on clinical trials utilizing adeno-associated virus and lentiviral vectors.
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“AAV vectors support long-term, non-toxic gene expression for up to 8 years in non-human primates.1–3”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [2]
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Self-citationAAV-based in vivo gene therapy for neurological disorders
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Metadata
- Authors
- Kantor, B., McCown, T., Leone, P., & Gray, S. J.
- Affiliations
- University of South Carolina (US)
- University of North Carolina at Chapel Hill (US)
- Rowan University (US)
- Corresponding author
- Gray, Steven J.
- Venue
- Advances in Genetics
- Publisher
- Elsevier
- Published
- 2014
- Volume
- 87
- Pages
- 71-124
- ISSN
- 0065-2660 (ISSN-L)
- PMC
- PMC4518844
- OpenAlex
- W2214048374
- Cited by
- 74
- References
- 216
- Citations by year
- 2026: 4 · 2025: 6 · 2024: 4 · 2023: 8 · 2022: 2 · 2021: 5 · 2020: 6 · 2019: 6 · 2018: 10 · 2017: 5 · 2016: 10 · 2015: 7 · 2014: 1
- FWCI
- 19.65
- Type
- book chapter
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Advances in genetics (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institute of Neurological Disorders and Stroke (P30NS045892, R01NS087175)
- 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
- central nervous system gene therapy, adeno-associated virus vectors, lentiviral vectors, blood-brain barrier, gene replacement therapy, Parkinson's disease, disease-modifying therapy, clinical trials
- MeSH terms
- Dependovirus, Central Nervous System Diseases, Genetic Vectors, Humans, Genetic Therapy, Lentivirus
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