ArticleNature biotechnology2017
A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear.
Article in Nature biotechnology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 211 papers, 1 of them a synthesis that pooled it.
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
211 citing papers in PubMed, 1 synthesis or guideline pooled it, 307 citations in OpenAlex.
- Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies.Frontiers in cellular neuroscience · 2021Pooled it
- AAV gene therapy for autosomal recessive deafness 9: a single-arm trial.Nature medicine · 2025Trial
- Efficient Endolymphatic Sac-Directed Gene Delivery Using AAV8BP2 and Posterior Semicircular Canal Injection.International journal of molecular sciences · 2026Article
- Rescue of stereocilia architecture and hearing function by AAV-CIB2 and AAV-CIB3 in a mouse model of recessive deafness DFNB48.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Review
- Age outweighs route for AAV-PHP.eB transgene distribution via inner ear delivery.Drug delivery and translational research · 2026Article
- Article
- Vestibular schwannoma: genetic and epigenetic mechanisms, hearing loss, and emerging therapies.Journal of neuro-oncology · 2026Review
- Human promoter-driven AAV tools enable precision gene therapy targeting cochlear hair cells.Cell reports. Medicine · 2026Article
- Postnatal Slc26a4 gene therapy improves hearing and structural integrity in a hereditary hearing loss model.The Journal of clinical investigation · 2026Article
- Pathogenic mechanism of the PLS1 gene variant in hearing loss and functional validation in a zebrafish model.Scientific reports · 2026Article
- Capsid-engineered AAV vector overcomes a key intracellular barrier and efficiently transduces spiral ganglion neurons in adult mice.Molecular therapy. Advances · 2026Article
- An engineered ferritin nanocage co-delivery system for targeted hair cells protection and hearing loss attenuation.Theranostics · 2026Article
- Hippo-YAP/TAZ signaling in inner ear development, injury, and regeneration: evidence synthesis and the biomechanical brake hypothesis.Frontiers in cell and developmental biology · 2026Review
- Cell-specific delivery of GJB2 restores auditory function in mouse models of DFNB1 deafness and mediates appropriate expression in NHP cochlea.Nature communications · 2025Article
- Recent preclinical and clinical advances in gene therapy for hereditary hearing loss.Molecules and cells · 2025Review
- ShH10 viral vector for safe, efficient, and selective transduction of inner ear supporting cells.Developmental biology · 2025Article
- Prevention of aminoglycoside-induced outer hair cell loss by silencing CaMKKβ in an acute mouse model.Hearing research · 2025Article
- Magnetic Targeting of AAV Gene Therapy for Inner Ear Following Systemic Delivery: Preliminary Findings and Transduction Pattern in Rat Cochlea.Journal of the Association for Research in Otolaryngology : JARO · 2025Article
- Inner Ear Organoid as a Preclinical Model of Hearing Regeneration: Progress and Applications.Stem cell reviews and reports · 2025Review
151 more citing papers are in PubMed but not listed here.
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Authors and funding
11 authors at 3 institutions in 3 countries.
Funding
Abstract
Efforts to develop gene therapies for hearing loss have been hampered by the lack of safe, efficient, and clinically relevant delivery modalities. Here we demonstrate the safety and efficiency of Anc80L65, a rationally designed synthetic vector, for transgene delivery to the mouse cochlea. Ex vivo transduction of mouse organotypic explants identified Anc80L65 from a set of other adeno-associated virus (AAV) vectors as a potent vector for the cochlear cell targets. Round window membrane injection resulted in highly efficient transduction of inner and outer hair cells in mice, a substantial improvement over conventional AAV vectors. Anc80L65 round window injection was well tolerated, as indicated by sensory cell function, hearing and vestibular function, and immunologic parameters. The ability of Anc80L65 to target outer hair cells at high rates, a requirement for restoration of complex auditory function, may enable future gene therapies for hearing and balance disorders.
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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.