ArticleFrontiers in molecular neuroscience2020
Intramuscular Delivery of Gene Therapy for Targeting the Nervous System.
Article in Frontiers in molecular neuroscience, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
25 citing papers in PubMed, 29 citations in OpenAlex.
- Targeting the peripheral nervous system in adipose tissue with engineered AAV vectors.Cell reports methods · 2026Article
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- In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression.Genome medicine · 2026Article
- Enterovirus D68 VP1 and VP3 determine neurotropism in human spinal cord organoids.bioRxiv : the preprint server for biology · 2026Article
- Nanocarrier imaging at single-cell resolution across entire mouse bodies with deep learning.Nature biotechnology · 2025Article
- AAV-based gene delivery of antimicrobial peptides to combat drug-resistant pathogens.Applied and environmental microbiology · 2025Review
- Safety and Immunogenicity of Recombinant Adeno-Associated Virus-Vectored African Swine Fever Virus Antigens.Veterinary medicine international · 2025Article
- Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside.Journal of translational medicine · 2024Review
- Targeting muscle to treat Charcot-Marie-Tooth disease.Neural regeneration research · 2024Article
- Viral-mediated gene therapy in pediatric neurological disorders.World journal of pediatrics : WJP · 2024Review
- Gene replacement therapies for inherited disorders of neurotransmission: Current progress in succinic semialdehyde dehydrogenase deficiency.Journal of inherited metabolic disease · 2024Review
- Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements.Molecular therapy. Methods & clinical development · 2024Review
- Micro/nanosystems for controllable drug delivery to the brain.Innovation (Cambridge (Mass.)) · 2024Review
- Virus-Based Biological Systems as Next-Generation Carriers for the Therapy of Central Nervous System Diseases.Pharmaceutics · 2023Review
- Vectored immunoprophylaxis and treatment of SARS-CoV-2 infection in a preclinical model.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- Editorial: Gene therapy for the central and peripheral nervous system, volume II.Frontiers in molecular neuroscience · 2023Article
- Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders.Journal of personalized medicine · 2022Review
- Editorial: Peripheral nerve anatomy in health and disease.Journal of anatomy · 2022Article
- Axon Biology in ALS: Mechanisms of Axon Degeneration and Prospects for Therapy.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2022Review
- Drug delivery to the central nervous system.Nature reviews. Materials · 2022Article
Corrections and comments
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Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
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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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.