Cited by 144Virus-based gene therapy researchRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

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The advent of AAV9 expands applications for brain and spinal cord gene delivery

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Dayton, Robert D, Wang, David B, Klein, Ronald L·2012·Expert Opinion on Biological Therapy

DOI 10.1517/14712598.2012.681463PMC PMC3361729

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Abstract

INTRODUCTION: Straightforward studies compared adeno-associated virus (AAV) serotypes to determine the most appropriate one for robust expression in the CNS. AAV9 was efficient when directly injected into the brain, but more surprisingly, AAV9 produced global expression in the brain and spinal cord after a peripheral, systemic route of administration to neonatal mice. AREAS COVERED: Topics include AAV9 gene delivery from intraparenchymal, intravenous, intrathecal and intrauterine routes of administration, and related preclinical studies and disease models. Systemic AAV9 gene transfer yields remarkably consistent neuronal expression, though only in early development. AAV9 is versatile to study neuropathological proteins: microtubule-associated protein tau and transactive response DNA-binding protein 43 kDa (TDP-43). EXPERT OPINION: AAV9 will be more widely used based on current data, although other natural serotypes and recombineered vectors may also support or improve upon wide-scale expression. A peripheral-to-central gene delivery that can affect the entire CNS without having to inject the CNS is promising for basic functional experiments, and potentially for gene therapy. Systemic or intra-cerebrospinal fluid routes of AAV9 administration should be considered for spinal muscular atrophy, lysosomal storage diseases and amyotrophic lateral sclerosis, if more neuronal expression can be achieved in adults, or if glial expression can be exploited.

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1 retracted

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Research works printed but not linked: 9

Lentz TB, 2011, Neurobiol Dis
Samaranch L, 2011, Hum Gene Ther
Federici T, 2011, Gene Ther
Mattar CN, 2012, Gene Ther
Nizzardo M, 2011, Cell Mol Life Sci
Federici T, 2011, Neurobiol Dis
Decressac M, 2012, Exp Neurol
Uchida A, 2012, Brain
Lin Y, 2011, Pain Res Treat

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“Gene therapy using AAV-FGF2 is similar in these regards, however FGL can be administered systemically and crosses the BBB, whereas AAV must be injected directly into the CNS, excluding AAV9 (Dayton et al. 2012).”
A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases
Körbelin, J., Dogbevia, G., Michelfelder, S., Ridder, D. A., Hunger, A., Wenzel, J., Seismann, H., Lampe, M., Bannach, J., Pasparakis, M., et al. (2016) · EMBO Molecular Medicine · doi:10.15252/emmm.201506078 · Cited by 253
“…et al, 2000; Cearley & Wolfe, 2006; de Backer et al, 2010; Markakis et al, 2010; Shen et al, 2013), and some of them (e.g., AAV9, AAVrh.8, and AAVrh.10) seem to be able to cross the mature BBB after systemic administration in adult animals (Foust et al, 2009; Dayton et al, 2012; Yang et al, 2014).”
Background
Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound
Thévenot, E., Jordão, J. F., O’Reilly, M. A., Markham, K. A., Weng, Y., Foust, K. D., Kaspar, B. K., Hynynen, K., & Aubert, I. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.013 · Cited by 202
“Advances in the AAV field have led to the selection of appropriate serotypes, exhibiting tropism for various organs (Pulicherla et al., 2011), and to the characterization of conditions that are important for infection efficiency (Gray et al., 2011; Dayton et al., 2012; Rapti et al., 2012).”
Background

See all 144

Authors: Dayton, R. D., Wang, D. B., & Klein, R. L.
Affiliations: Louisiana State University Health Sciences Center Shreveport (US)
University of Washington (US)
Corresponding author: Klein, Ronald L
ORCID: Dayton, Robert D
Venue: Expert Opinion on Biological Therapy
Publisher: Informa Healthcare
Published: 2012-04-20
Volume: 12
Issue: 6
Pages: 757-766
ISSN: 1471-2598 (ISSN-L)
1744-7682
DOI: 10.1517/14712598.2012.681463
PMC: PMC3361729
OpenAlex: W2109349667
Cited by: 144
References: 75
Citations by year: 2026: 7 · 2025: 13 · 2024: 11 · 2023: 13 · 2022: 8 · 2021: 4 · 2020: 7 · 2019: 7 · 2018: 8 · 2017: 9 · 2016: 14 · 2015: 12 · 2014: 14 · 2013: 13 · 2012: 4
FWCI: 1.68
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Locations: Expert Opinion on Biological Therapy (publishedVersion)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
Funders: National Institute of Neurological Disorders and Stroke (R01NS048450, NS048450)
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
Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
Keywords: AAV9, adeno-associated virus serotypes, intrathecal administration, intravenous administration, intraparenchymal injection, intrauterine administration, neonatal mice, neuronal expression, tau protein, TDP-43, spinal muscular atrophy, lysosomal storage disease, amyotrophic lateral sclerosis, glial expression, gene therapy vectors
MeSH terms: Dependovirus, Animals, Brain, Gene Expression Regulation, Genetic Vectors, Humans, Injections, Intravenous, Injections, Intraventricular, Injections, Spinal, Neurons, Spinal Cord, Genetic Therapy, Gene Transfer Techniques

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The advent of AAV9 expands applications for brain and spinal cord gene delivery

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