ArticleMolecular therapy : the journal of the American Society of Gene Therapy2015
Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 1 of them a synthesis that pooled it.
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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
28 citing papers in PubMed, 1 synthesis or guideline pooled it, 47 citations in OpenAlex.
- Optogenetic control of the enteric nervous system and gastrointestinal transit.Expert review of gastroenterology & hepatology · 2019Pooled it
- Gene Therapy in Crohn's Disease: Current Preclinical Challenges and Future Translational Avenues.Biomedicines · 2025Review
- Colorectal cancer cells hijack a brain-gut polysynaptic circuit from the lateral septum to enteric neurons to sustain tumor growth.Nature cancer · 2025Article
- Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models.Science translational medicine · 2025Article
- Protective effects of medicinal plant-derived metabolites on slow transit constipation via the ENS-ICC-SMC pathway.Frontiers in pharmacology · 2025Review
- Recent developments in gene therapy research in India.Journal of biosciences · 2024Review
- Adeno-associated virus-9 reverses delayed gastric emptying of solids in diabetic mice.Neurogastroenterology and motility · 2023Article
- Stimulator of interferon genes (STING) expression in the enteric nervous system and contributions of glial STING in disease.Neurogastroenterology and motility · 2023Article
- Adeno-associated virus vector intraperitoneal injection induces colonic mucosa and submucosa transduction and alters the diversity and composition of the faecal microbiota in rats.Frontiers in cellular and infection microbiology · 2022Article
- Transduction of Systemically Administered Adeno-Associated Virus in the Colonic Enteric Nervous System and c-Kit Cells of Adult Mice.Frontiers in neuroanatomy · 2022Article
- Epigenetic inactivation of the autophagy-lysosomal system in appendix in Parkinson's disease.Nature communications · 2021Article
- Retinal Tropism and Transduction of Adeno-Associated Virus Varies by Serotype and Route of Delivery (Intravitreal, Subretinal, or Suprachoroidal) in Rats.Human gene therapy · 2020Article
- AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy.Frontiers in molecular neuroscience · 2020Review
- Optogenetic analysis of neuromuscular transmission in the colon of ChAT-ChR2-YFP BAC transgenic mice.American journal of physiology. Gastrointestinal and liver physiology · 2019Article
- AAV-Mediated Gene Delivery to the Enteric Nervous System by Intracolonic Injection.Methods in molecular biology (Clifton, N.J.) · 2019Article
- Induction of alpha-synuclein pathology in the enteric nervous system of the rat and non-human primate results in gastrointestinal dysmotility and transient CNS pathology.Neurobiology of disease · 2018Article
- Optogenetic and chemogenetic techniques for neurogastroenterology.Nature reviews. Gastroenterology & hepatology · 2018Review
- Introducing a Novel Method of Intravascular Adeno-associated Virus-mediated Gene Delivery.Virology (Hyderabad) · 2018Article
- Systemic gene delivery transduces the enteric nervous system of guinea pigs and cynomolgus macaques.Gene therapy · 2017Article
- Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS.Molecular therapy. Nucleic acids · 2017Article
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
Abstract
Recombinant adeno-associated virus (AAV) vectors are one of the most widely used gene transfer systems in research and clinical trials. AAV can transduce a wide range of biological tissues, however to date, there has been no investigation on targeted AAV transduction of the enteric nervous system (ENS). Here, we examined the efficiency, tropism, spread, and immunogenicity of AAV transduction in the ENS. Rats received direct injections of various AAV serotypes expressing green fluorescent protein (GFP) into the descending colon. AAV serotypes tested included; AAV 1, 2, 5, 6, 8, or 9 and the AAV2 and AAV8 capsid mutants, AAV2-Y444F, AAV2-tripleY-F, AAV2-tripleY-F+T-V, AAV8-Y733F, and AAV8-doubeY-F+T-V. Transduction, as determined by GFP-positive cells, occurred in neurons and enteric glia within the myenteric and submucosal plexuses of the ENS. AAV6 and AAV9 showed the highest levels of transduction within the ENS. Transduction efficiency scaled with titer and time, was translated to the murine ENS, and produced no vector-related immune response. A single injection of AAV into the colon covered an area of ~47 mm(2). AAV9 primarily transduced neurons, while AAV6 transduced enteric glia and neurons. This is the first report on targeted AAV transduction of neurons and glia in the ENS.
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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.