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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IM delivery strategy is particularly suited for mitigating motor neuron diseases, such as spinal muscular atrophy and amyotrophic lateral sclerosis.
“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]
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.
“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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Multiple retrograde tracing methods compatible with 3DISCO clearing
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α CAR IGF ‐1 vector targeting of motor neurons ameliorates disease progression in ALS mice
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Flagged first, then by citations
1 of the 205 references couldn’t be matched to a known paper.
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Drug delivery to the central nervous system
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“Intramuscular delivery of viral vectors for gene therapy could be an effective way to deliver such therapies to distal motor axons, in addition to delivering therapies to the soma via retrograde axonal transport [111].”
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Neuroanatomical Tract Tracers: Not Just for Neural Tracing Anymore
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Viral-mediated gene therapy in pediatric neurological disorders
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Planet of the AAVs: The Spinal Cord Injury Episode
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Vectored immunoprophylaxis and treatment of SARS-CoV-2 infection in a preclinical model
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AAV-based gene delivery of antimicrobial peptides to combat drug-resistant pathogens
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Targeting muscle to treat Charcot-Marie-Tooth disease
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Recent Scientific Breakthroughs Applying CRISPR Gene Editing in Neurological Disorders
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Editorial: Peripheral nerve anatomy in health and disease
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Self-citationGene therapy in neurodegenerative disorders
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In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression
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Vectored Immunoprophylaxis and Treatment of SARS-CoV-2 InfectionPreprint
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Enterovirus D68 VP1 and VP3 determine neurotropism in human spinal cord organoidsPreprint
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Self-citationTargeting the peripheral nervous system in adipose tissue with engineered AAV vectors
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Advances in the development and delivery of nucleic acid-based therapeutics
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- 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 › 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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