ArticleThe Journal of clinical investigation2009
DRG-targeted helper-dependent adenoviruses mediate selective gene delivery for therapeutic rescue of sensory neuronopathies in mice.
Article in The Journal of clinical investigation, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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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
24 citing papers in PubMed, 35 citations in OpenAlex.
- Peptide-Incorporated Biomaterials Promote Regeneration of Peripheral Nerve Injuries.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Review
- Pro-inflammatory microglia-targeted peptide therapy ameliorates neonatal hypoxic-ischemic encephalopathy in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Sensory-motor circuit is a therapeutic target forScience advances · 2024Article
- Rapid injection of lumbar dorsal root ganglia under direct vision: Relevant anatomy, protocol, and behaviors.Frontiers in neurology · 2023Article
- Bone-Marrow-Derived Mononuclear Cells Relieve Neuropathic Pain after Spinal Nerve Injury in Mice.Molecular therapy. Methods & clinical development · 2020Article
- High-Capacity Adenoviral Vectors: Expanding the Scope of Gene Therapy.International journal of molecular sciences · 2020Review
- TFEB regulates murine liver cell fate during development and regeneration.Nature communications · 2020Article
- Pre-arrayed Pan-AAV Peptide Display Libraries for Rapid Single-Round Screening.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Intramuscular Delivery of Gene Therapy for Targeting the Nervous System.Frontiers in molecular neuroscience · 2020Article
- Advanced Technology for Gene Delivery with Homing Peptides to Spinal Cord through Systemic Circulation in Mice.Molecular therapy. Methods & clinical development · 2019Article
- Efficient Prostate Cancer Therapy with Tissue-Specific Homing Peptides Identified by Advanced Phage Display Technology.Molecular therapy oncolytics · 2019Article
- Gene therapy for neuropathic pain using dorsal root ganglion-targeted helper-dependent adenoviral vectors with GAD67 expression.Pain reports · 2018Article
- Gene Therapy for Neuropathic Pain through siRNA-IRF5 Gene Delivery with Homing Peptides to Microglia.Molecular therapy. Nucleic acids · 2018Article
- Noncoding RNAs: key molecules in understanding and treating pain.Trends in molecular medicine · 2014Review
- DNA cleavage enzymes for treatment of persistent viral infections: recent advances and the pathway forward.Virology · 2014Review
- Gene therapy for neuropathic pain by silencing of TNF-α expression with lentiviral vectors targeting the dorsal root ganglion in mice.PloS one · 2014Article
- Safety profile, efficacy, and biodistribution of a bicistronic high-capacity adenovirus vector encoding a combined immunostimulation and cytotoxic gene therapy as a prelude to a phase I clinical trial for glioblastoma.Toxicology and applied pharmacology · 2013Article
- Overexpression of endothelial nitric oxide synthase improves endothelium-dependent vasodilation in arteries infused with helper-dependent adenovirus.Human gene therapy · 2012Article
- Safety profile of gutless adenovirus vectors delivered into the normal brain parenchyma: implications for a glioma phase 1 clinical trial.Human gene therapy methods · 2012Article
- Dorsal root ganglion - a potential new therapeutic target for neuropathic pain.Journal of pain research · 2012Article
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 2 countries.
Funding
Abstract
Dorsal root ganglion (DRG) neuron dysfunction occurs in a variety of sensory neuronopathies for which there are currently no satisfactory treatments. Here we describe the development of a strategy to target therapeutic genes to DRG neurons for the treatment of these disorders. We genetically modified an adenovirus (Ad) to generate a helper virus (HV) that was detargeted for native adenoviral tropism and contained DRG homing peptides in the adenoviral capsid fiber protein; we used this HV to generate DRG-targeted helper-dependent Ad (HDAd). In mice, intrathecal injection of this HDAd produced a 100-fold higher transduction of DRG neurons and a markedly attenuated inflammatory response compared with unmodified HDAd. We also injected HDAd encoding the beta subunit of beta-hexosaminidase (Hexb) into Hexb-deficient mice, a model of the neuronopathy Sandhoff disease. Delivery of the DRG-targeted HDAd reinstated neuron-specific Hexb production, reversed gangliosidosis, and ameliorated peripheral sensory dysfunction. The development of DRG neuron-targeted HDAd with proven efficacy in a preclinical model may have implications for the treatment of sensory neuronopathies of diverse etiologies.
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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.