Cited by 74Virus-based gene therapy researchRNA Interference and Gene DeliveryBarrier Structure and Function Studies

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Clinical Applications Involving CNS Gene Transfer

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Kantor, Boris, McCown, Thomas, Leone, Paola, Gray, Steven J.·2014·Advances in Genetics

DOI 10.1016/b978-0-12-800149-3.00002-0PMC PMC4518844

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Abstract

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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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.
ORCID: Kantor, Boris
McCown, Thomas
Leone, Paola
Gray, Steven J.
Venue: Advances in Genetics
Publisher: Elsevier
Published: 2014
Volume: 87
Pages: 71-124
ISSN: 0065-2660 (ISSN-L)
DOI: 10.1016/b978-0-12-800149-3.00002-0
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
Licence: www.elsevier.com/tdm/userlicense/1.0/ (tdm)
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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