The DOI matches its Crossref record; 25 of the 25 references it cites were found in a registry or an index.
Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6
Open accessAbstract
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.
Overview
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
In the citing paper
“The vectors stay predominantly in the lower spine in intrathecal delivery administrations.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [93]
In the citing paper
“Use of intrathecal delivery for gene therapy vectors has been limited to studies involving spinal cord and peripheral nerve disease.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [93]
In the citing paper
“Moreover, IM delivery of AAV6 showed about 50% transduction of spinal motor neurons in NHPs [105].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [105]
Citations
Cites
Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163
Gene therapy using adeno-associated virus vectors
Daya, S., & Berns, K. I. (2008) · Clinical Microbiology Reviews · doi:10.1128/cmr.00008-08 · Cited by 926
Retrograde Viral Delivery of IGF-1 Prolongs Survival in a Mouse ALS Model
Kaspar, B. K., Lladó, J., Sherkat, N., Rothstein, J. D., & Gage, F. H. (2003) · Science · doi:10.1126/science.1086137 · Cited by 872
Ralph, G. S., Radcliffe, P. A., Day, D. M., Carthy, J. M., Leroux, M. A., Lee, D. C. P., Wong, L., Bilsland, L. G., Greensmith, L., Kingsman, S. M., et al. (2005) · Nature Medicine · doi:10.1038/nm1205 · Cited by 507
Incidence, prevalence, and gene frequency studies of chronic childhood spinal muscular atrophy.
Pearn, J. H. (1978) · Journal of Medical Genetics · doi:10.1136/jmg.15.6.409 · Cited by 475
Grimm, D., Kay, M. A., & Kleinschmidt, J. A. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00095-9 · Cited by 344
Wu, Z., Asokan, A., Grieger, J. C., Lakshmanan Govindasamy, A., Agbandje‐McKenna, M., & Samulski, R. J. (2006) · Journal of Virology · doi:10.1128/jvi.01288-06 · Cited by 239
Peden, C. S., Burger, C., Muzyczka, N., & Mandel, R. J. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6344-6359.2004 · Cited by 222
Wang, Z., Kuhr, C. S., Allen, J. M., Blankinship, M. J., Gregorevic, P., Chamberlain, J. S., Tapscott, S. J., & Storb, R. F. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300161 · Cited by 220
Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy
Azzouz, M., Le, T. T., Ralph, G. S., Walmsley, L. E., Monani, U. R., Lee, D. C. P., Wilkes, F. J., Mitrophanous, K. A., Kingsman, S. M., Burghes, A. H. M., et al. (2004) · Journal of Clinical Investigation · doi:10.1172/jci22922 · Cited by 209
Flagged first, then by citations
Cited by
Sorted by citations
Items 101–124 of 124
Gene Delivery and Gene Therapy for Alzheimer’s Disease
Woodbury, M. E., Kiyota, T., & Ikezu, T. (2015) · Neuromethods · doi:10.1007/978-1-4939-2306-9_4 · Cited by 2
Williams, J. J., Vazquez, A. L., & Schwartz, A. B. (2019) · International IEEE/EMBS Conference on Neural Engineering · doi:10.1109/ner.2019.8716978 · Cited by 2
Mir, M. Y., Seh, B. A., Rafiq, K., & Légrádi, Á. (2026) · International Journal of Molecular Sciences · doi:10.3390/ijms27062845 · Cited by 1
Ghosh, U., & Tay, A. K. P. (2026) · Journal of Controlled Release · doi:10.1016/j.jconrel.2026.115204 · Cited by 1
AAV6-Mediated Gene Silencing fALS Short
Mandel, R. J., Lowenstein, P. R., & Byrne, B. J. (2011) · Molecular Therapy · doi:10.1038/mt.2010.306 · Cited by 1
Current and investigational treatments for spinal muscular atrophy
Tam, J. K. V., Karyka, E., & Azzouz, M. (2014) · Expert Opinion on Orphan Drugs · doi:10.1517/21678707.2014.901910 · Cited by 1
The Use of Viral Vectors to Enhance Cognition
Ikezu, T. (2015) · Elsevier eBooks · doi:10.1016/b978-0-12-417042-1.00006-1 · Cited by 1
Gene Therapy for Spinal Cord Injury
Goganau, I., & Blesch, A. (2016) · Springer eBooks · doi:10.1007/978-1-4899-7654-3_8 · Cited by 1
López-García, M. Á., Feria-Romero, I. A., Segura-Uribe, J. J., Escalante-Santiago, D., & Orozco-Suárez, S. A. (2016) · Methods in pharmacology and toxicology · doi:10.1007/978-1-4939-6355-3_10 · Cited by 1
Gene Therapy for Neurological Diseases
Simonato, M., Wahlberg, L. U., Goins, W. F., & Glorioso, J. C. (2015) · Elsevier eBooks · doi:10.1016/b978-0-12-800563-7.00009-9 · Cited by 1
Development of Therapies for Spinal Muscular Atrophy Using Gene Therapy and Nanotechnology
Little, D. (2013) · White Rose eTheses Online (University of Leeds, The University of Sheffield, University of York)
Organization and connectivity of premotor interneurons in the mouse spinal cord
Stepien, A. E. (2011) · edoc (University of Basel) · doi:10.5451/unibas-005712076
Davis, A. (2012) · OhioLink ETD Center (Ohio Library and Information Network)
Gene Therapy for Motor Neuron Disease
Boulis, N. M. (2011)
Application of Viral Vectors to Motor Neuron Disorders
Dirren, E., & Schneider, B. L. (2013) · Neuromethods · doi:10.1007/978-1-62703-610-8_12
Self-citationPeripheral mechanisms of touch and pain
Sexton, J. (2016) · UCL Discovery (University College London)
Therapeutic strategies for spinal muscular atrophy: looking beyond the SMN gene
Bowerman, M., Becker, C. G., Yáñez‐Muñoz, R. J., Ning, K., Wood, M. J. A., Gillingwater, T. H., & Talbot, K. (2017) · Disease Models & Mechanisms
Viral vector gene therapy approaches for regeneration and repair in spinal cord injury
O’Carroll, S. J., & Clemett, C. A. (2022) · Elsevier eBooks · doi:10.1016/b978-0-12-822498-4.00032-4
Virus-mediated neuron-specific retrograde gene delivery in spinal cord injury
Chelyshev, Y. A., & Mukhamedshina, Y. O. (2024) · Genes and Cells · doi:10.17816/gc624407
A non-human primate model of Amyotrophic Lateral SclerosisPreprint
Jones, R. H., Saieva, L., Balezeau, F., Schofield, I. S., McCardle, C., Baker, M., & Baker, S. N. (2025) · eLife · doi:10.1101/2025.10.27.684759
Aebischer, J. (2011) · theses.fr (ABES) · doi:10.70675/7dc30612zb4b4z49a3z864bz8fa2e995aa0e
Développement de nouveaux modèles expérimentaux de la Sclérose Latérale Amyotrophique
Langou, K. (2010) · theses.fr (ABES) · doi:10.70675/b77c5438zccd7z4f8bz935az7ab30698b8a8
Studying Neuronal Connectivity in the Mouse Brain in Normal Condition and Fragile X Syndrome
Haberl, M. G. (2014) · theses.fr (ABES) · doi:10.70675/cbb47168z82fbz41a1z9174zd9e36b2b5a6d
Journou, M. (2021) · theses.fr (ABES) · doi:10.70675/9a618ac2za0a4z4e90zbdb3ze14482ffa655
Metadata
- 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
- Published
- 2009-09-03
- Volume
- 17
- Issue
- 1
- Pages
- 141-146
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- 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
- 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
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
