ArticleHuman gene therapy methods2012
Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9.
Article in Human gene therapy methods, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
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Who cites it
35 citing papers in PubMed, 44 citations in OpenAlex.
- Preparation and application of YFV-17D-derived Anterograde Trans-neuronal Viral Vectors for Neuroscience Research.bioRxiv : the preprint server for biology · 2026Article
- GAA replacement improves respiratory muscle, neural, and alveolar pathology in the pompe mouse.Respiratory physiology & neurobiology · 2025Article
- Optogenetic activation of the tongue in spontaneously breathing mice.Respiratory physiology & neurobiology · 2023Article
- Targeting drug or gene delivery to the phrenic motoneuron pool.Journal of neurophysiology · 2023Review
- Chemogenetic activation of hypoglossal motoneurons in a mouse model of Pompe disease.Journal of neurophysiology · 2022Article
- Article
- Gene delivery to the hypoglossal motor system: preclinical studies and translational potential.Gene therapy · 2021Review
- AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A.Nature communications · 2021Article
- Tongue and hypoglossal morphology after intralingual cholera toxin B-saporin injection.Muscle & nerve · 2021Article
- Respiratory pathology in the OptnRespiratory physiology & neurobiology · 2020Article
- Intralingual Administration of AAVrh10-miRHuman gene therapy · 2020Article
- Intralingual and Intrapleural AAV Gene Therapy Prolongs Survival in a SOD1 ALS Mouse Model.Molecular therapy. Methods & clinical development · 2020Article
- AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice.Molecular therapy. Methods & clinical development · 2019Article
- Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy.Annals of translational medicine · 2019Review
- Progress and challenges of gene therapy for Pompe disease.Annals of translational medicine · 2019Review
- Hypoglossal Motor Neuron Death Via Intralingual CTB-saporin (CTB-SAP) Injections Mimic Aspects of Amyotrophic Lateral Sclerosis (ALS) Related to Dysphagia.Neuroscience · 2018Article
- A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.Molecular neurobiology · 2018Article
- The impact of Pompe disease on smooth muscle: a review.Journal of smooth muscle research = Nihon Heikatsukin Gakkai kikanshi · 2018Review
- Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice.Respiratory physiology & neurobiology · 2016Article
- Power spectral analysis of hypoglossal nerve activity during intermittent hypoxia-induced long-term facilitation in mice.Journal of neurophysiology · 2016Article
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Authors and funding
9 authors at 2 institutions in 1 country.
Funding
Abstract
Retrograde viral transport (i.e., muscle to motoneuron) enables targeted gene delivery to specific motor pools. Recombinant adeno-associated virus serotype 9 (AAV9) robustly infects motoneurons, but the retrograde transport capabilities of AAV9 have not been systematically evaluated. Accordingly, we evaluated the retrograde transduction efficiency of AAV9 after direct tongue injection in 129SVE mice as well as a mouse model that displays neuromuscular pathology (Gaa(-/-)). Hypoglossal (XII) motoneurons were histologically evaluated 8 weeks after tongue injection with AAV9 encoding green fluorescent protein (GFP) with expression driven by the chicken β-actin promoter (1 × 10(11) vector genomes). On average, GFP expression was detected in 234 ± 43 XII motoneurons 8 weeks after AAV9-GFP tongue injection. In contrast, tongue injection with a highly efficient retrograde anatomical tracer (cholera toxin β subunit, CT-β) resulted in infection of 818 ± 88 XII motoneurons per mouse. The retrograde transduction efficiency of AAV9 was similar between the 129SVE mice and those with neuromuscular disease (Gaa(-/-)). Routine hematoxylin and eosin staining and cluster of differentiation (CD) immunostaining for T cells (CD3) indicated no persistent inflammation within the tongue or XII nucleus after AAV9 injection. Additional experiments indicated no adverse effects of AAV9 on the pattern of breathing. We conclude that AAV9 can retrogradely infect a significant portion of a given motoneuron pool in normal and dystrophic mice, and that its transduction efficiency is approximately 30% of what can be achieved with CT-β.
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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.