Methods for Gene Transfer to the Central Nervous System

Kantor, Boris; Bailey, Rachel M.; Wimberly, Keon; Kalburgi, Sahana N.; Gray, Steven J.

doi:10.1016/b978-0-12-800149-3.00003-2

Cited by 109Virus-based gene therapy researchRNA Interference and Gene DeliveryBarrier Structure and Function Studies

Methods for Gene Transfer to the Central Nervous System

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Kantor, Boris, Bailey, Rachel M., Wimberly, Keon, Kalburgi, Sahana N., Gray, Steven J.·2014·Advances in Genetics

DOI 10.1016/b978-0-12-800149-3.00003-2PMC PMC4519829

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Abstract

Gene transfer is an increasingly utilized approach for research and clinical applications involving the central nervous system (CNS). Vectors for gene transfer can be as simple as an unmodified plasmid, but more commonly involve complex modifications to viruses to make them suitable gene delivery vehicles. This chapter will explain how tools for CNS gene transfer have been derived from naturally occurring viruses. The current capabilities of plasmid, retroviral, adeno-associated virus, adenovirus, and herpes simplex virus vectors for CNS gene delivery will be described. These include both focal and global CNS gene transfer strategies, with short- or long-term gene expression. As is described in this chapter, an important aspect of any vector is the cis-acting regulatory elements incorporated into the vector genome that control when, where, and how the transgene is expressed.

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AAV vectors support long-term, non-toxic gene expression for up to 8 years in non-human primates.
“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 [3]
Section 2.2 (page not provided) reports AAV-delivered expression lasting at least 6 years in primates; it assigns the 8-year figure to dogs, not primates. The same section describes AAV vectors as having no known associated pathologies and causing a very mild immune response, which supports the safety characterization but does not establish an eight-year non-toxic outcome in primates. (LLM, conf=0.90)
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Authors: Kantor, B., Bailey, R. M., Wimberly, K., Kalburgi, S. N., & Gray, S. J.
Affiliations: University of South Carolina (US)
University of North Carolina at Chapel Hill (US)
Corresponding author: Gray, Steven J.
ORCID: Kantor, Boris
Bailey, Rachel M.
Wimberly, Keon
Kalburgi, Sahana N.
Gray, Steven J.
Venue: Advances in Genetics
Publisher: Elsevier
Published: 2014
Volume: 87
Pages: 125-197
ISSN: 0065-2660 (ISSN-L)
DOI: 10.1016/b978-0-12-800149-3.00003-2
PMC: PMC4519829
OpenAlex: W2414165069
Cited by: 109
References: 338
Citations by year: 2026: 3 · 2025: 11 · 2024: 9 · 2023: 9 · 2022: 10 · 2021: 17 · 2020: 9 · 2019: 6 · 2018: 11 · 2017: 9 · 2016: 14 · 2015: 1
FWCI: 20.49
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: gene delivery vectors, plasmid vectors, retroviral vectors, adeno-associated virus vectors, adenoviral vector, herpes simplex virus vectors, cis-regulatory elements, transgene expression
MeSH terms: Adenoviridae, Central Nervous System, Genetic Vectors, Humans, Genetic Therapy, Gene Expression, Lentivirus, Gene Transfer Techniques, Simplexvirus

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Methods for Gene Transfer to the Central Nervous System

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