The DOI matches its Crossref record; 204 of the 205 references it cites were found in a registry or an index.
Intramuscular Delivery of Gene Therapy for Targeting the Nervous System
Open accessAbstract
Virus-mediated gene therapy has the potential to deliver exogenous genetic material into specific cell types to promote survival and counteract disease. This is particularly enticing for neuronal conditions, as the nervous system is renowned for its intransigence to therapeutic targeting. Administration of gene therapy viruses into skeletal muscle, where distal terminals of motor and sensory neurons reside, has been shown to result in extensive transduction of cells within the spinal cord, brainstem, and sensory ganglia. This route is minimally invasive and therefore clinically relevant for gene therapy targeting to peripheral nerve soma. For successful transgene expression, viruses administered into muscle must undergo a series of processes, including host cell interaction and internalization, intracellular sorting, long-range retrograde axonal transport, endosomal liberation, and nuclear import. In this review article, we outline key characteristics of major gene therapy viruses-adenovirus, adeno-associated virus (AAV), and lentivirus-and summarize the mechanisms regulating important steps in the virus journey from binding at peripheral nerve terminals to nuclear delivery. Additionally, we describe how neuropathology can negatively influence these pathways, and conclude by discussing opportunities to optimize the intramuscular administration route to maximize gene delivery and thus therapeutic potential.
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“This strategy is particularly suited for mitigating motor neuron diseases (MNDs), such as spinal muscular atrophy (SMA) [99] and amyotrophic lateral sclerosis (ALS) [101].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [99]
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“AAV-mediated gene therapy using IM delivery is minimally invasive in local muscle tissue and undergoes a cascade intracellular event for retrograde axonal trafficking into neuronal soma [99, 100].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [99]
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Items 201–204 of 204
Bohlen, M. O., El-Nahal, H. G., & Sommer, M. A. (2019) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2019.00084 · Cited by 10
Differential regulation of axonal growth and neuromuscular junction assembly by HGF/c‐Met signaling
Li, P. P., Madhavan, R., & Peng, H. B. (2012) · Developmental Dynamics · doi:10.1002/dvdy.23845 · Cited by 7
Larochelle, N., Teng, Q., Gilbert, R., Deol, J. R., Karpati, G., Holland, P. C., & Nalbantoglu, J. N. (2010) · The Journal of Gene Medicine · doi:10.1002/jgm.1433 · Cited by 6
Zhao, Y., Haginoya, K., & Iinuma, K. (1999) · The Tohoku Journal of Experimental Medicine · doi:10.1620/tjem.189.239 · Cited by 6
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Nance, E. A., Pun, S. H., Saigal, R., & Sellers, D. L. (2021) · Nature Reviews Materials · doi:10.1038/s41578-021-00394-w · Cited by 442
Chowdhury, E. A., Meno‐Tetang, G. M. L., Chang, H. Y., Wu, S., Huang, H. W., Jamier, T., Chandran, J. S., & Shah, D. K. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2021.01.017 · Cited by 80
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Tian, M., Ma, Z., & Yang, G. (2023) · The Innovation · doi:10.1016/j.xinn.2023.100548 · Cited by 64
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Luo, J., Molbay, M., Chen, Y., Horvath, I., Kadletz, K., Kick, B., Zhao, S., Al-Maskari, R., Singh, I., Ali, M., et al. (2025) · Nature Biotechnology · doi:10.1038/s41587-024-02528-1 · Cited by 53
Xu, L., Yao, S., Ding, Y. E., Xie, M., Feng, D., Sha, P., Tan, L., Bei, F., & Yao, Y. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05661-2 · Cited by 48
Metadata
- Authors
- Tosolini, A. P., & Sleigh, J. N.
- Affiliations
- UCL Queen Square Institute of Neurology (GB)
- UK Dementia Research Institute (GB)
- Corresponding author
- Tosolini, Andrew P.
- Sleigh, James N.
- Venue
- Frontiers in Molecular Neuroscience
- Publisher
- Frontiers Media SA
- Published
- 2020-07-17
- Volume
- 13
- Pages
- 129-129
- ISSN
- 1662-5099 (ISSN-L)
- PMC
- PMC7379875
- OpenAlex
- W3043715250
- Cited by
- 32
- References
- 205
- Citations by year
- 2026: 4 · 2025: 4 · 2024: 5 · 2023: 8 · 2022: 5 · 2021: 6
- FWCI
- 2.79
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Frontiers in Molecular Neuroscience (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- UCL Discovery (University College London) (submittedVersion)
- Funders
- Wellcome Trust (107116/Z/15/Z)
- Medical Research Council (MR/S006990/1)
- 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
- intramuscular gene delivery, gene therapy vectors, retrograde axonal transport, peripheral nerve terminals, motor neurons, sensory neurons, adeno-associated virus, adenovirus, lentivirus, viral transduction, endosomal escape, nuclear import, neuropathology
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