Cited by 124Virus-based gene therapy researchNeurogenetic and Muscular Disorders ResearchRNA Interference and Gene Delivery

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Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6

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Towne, C, Schneider, B L, Kieran, D, Redmond, D E, Aebischer, P·2010·Gene Therapy

DOI 10.1038/gt.2009.119

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Abstract

Retrograde transport of viral vectors in the rodent spinal cord provides a powerful means to administer a therapeutic transgene from the innervated musculature. With the aim of scaling up this approach to non-human primates, we have injected recombinant adeno-associated vectors (rAAV) serotype 6 expressing enhanced green fluorescent protein (eGFP) into the gastrocnemius muscle of African green monkeys to determine whether this results in efficient transgene delivery to lumbar motor neurons. Cells expressing eGFP were observed across more than 1 cm of the spinal cord 4 weeks after intramuscular injection, reaching more than half of motor neurons in some cross-sections. Furthermore, quantitative PCR on the spinal cord tissue confirmed that eGFP expression within motor neurons was due to bona fide retrograde transport of the vector genome from the muscle. Although infiltrations of macrophages and lymphocytes were observed in the rAAV2/6-injected muscle, there was no detectable immune response within the transduced region of the spinal cord. These findings imply that retrograde delivery of rAAV serotype 6 in a primate species constitutes a non-invasive and robust approach to transduce motor neurons, a crucial target cell population in neurodegenerative disorders, such as amyotrophic lateral sclerosis and spinal muscular atrophy.

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Authors: Towne, C., Schneider, B. L., Kieran, D., Redmond, D. E., & Aebischer, P.
Affiliations: École Polytechnique Fédérale de Lausanne (CH)
Yale University (US)
Corresponding author: Aebischer, P
ORCID: Schneider, B L
Venue: Gene Therapy
Publisher: Springer Science and Business Media LLC
Published: 2009-09-03
Volume: 17
Issue: 1
Pages: 141-146
ISSN: 0969-7128 (ISSN-L)
1476-5462
DOI: 10.1038/gt.2009.119
OpenAlex: W2151102772
Cited by: 124
References: 25
Citations by year: 2026: 2 · 2025: 2 · 2024: 4 · 2023: 6 · 2022: 4 · 2021: 7 · 2020: 5 · 2019: 11 · 2018: 5 · 2017: 8 · 2016: 11 · 2015: 6 · 2014: 7 · 2013: 14 · 2012: 7
FWCI: 6.22
Type: journal article
Language: en
Open access: green
infoscience.epfl.ch
Licence: www.springer.com/tdm (tdm)
www.springer.com/tdm (vor)
Locations: Gene Therapy (publishedVersion)
PubMed (publishedVersion)
Infoscience (Ecole Polytechnique Fédérale de Lausanne) (submittedVersion)
Infoscience (Ecole Polytechnique Fédérale de Lausanne) (submittedVersion, cc-by-nc-nd)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Keywords: motor neurons, intramuscular injection, non-human primates, vervet monkey, spinal cord, retrograde transport, EGFP expression, neurodegenerative diseases, amyotrophic lateral sclerosis, spinal muscular atrophy, immune response
MeSH terms: Dependovirus, Animals, Chlorocebus aethiops, Genetic Vectors, Injections, Intramuscular, Motor Neurons, Spinal Cord, Transduction, Genetic, Transgenes, Green Fluorescent Proteins

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Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6

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