ArticleMolecular therapy : the journal of the American Society of Gene Therapy2017
Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 160 papers, 2 of them syntheses that pooled it.
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Who cites it
160 citing papers in PubMed, 2 syntheses or guidelines pooled it, 243 citations in OpenAlex.
- Research progress on exosomes in the otology field: a systematic review.Stem cell research & therapy · 2025Pooled it
- Transduction Efficiency and Immunogenicity of Viral Vectors for Cochlear Gene Therapy: A Systematic Review of Preclinical Animal Studies.Frontiers in cellular neuroscience · 2021Pooled it
- Exploration of biodistribution of mRNA-delivering lipid nanoparticles in the neonatal mouse inner ear.International journal of pharmaceutics: X · 2026Article
- Host determinants of exo-rAAV production revealed by extended gene expression analysis in HEK293 cells.Molecular therapy. Advances · 2026Article
- Efficient Endolymphatic Sac-Directed Gene Delivery Using AAV8BP2 and Posterior Semicircular Canal Injection.International journal of molecular sciences · 2026Article
- Exosomes from mesenchymal stem/stromal cells and their therapeutic role in osteoarthritic pain.Journal of experimental orthopaedics · 2026Article
- Distinguishing Pseudotransduction and True Transduction Enables Characterization and Bioengineering of Extracellular Vesicle-Adeno-Associated Virus Vectors.Journal of extracellular vesicles · 2026Article
- Exploiting the role of milk extracellular vesicles: a comprehensive analysis on isolation methods, characterization, surface modifications, and their therapeutic applications.Journal of nanobiotechnology · 2026Review
- Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Interactions between extracellular vesicles and viruses: lessons learned across species and kingdoms.FEMS microbiology reviews · 2026Review
- Engineered Exosomes: Advances in Therapeutic Applications for Otolaryngology-Head and Neck Diseases.International journal of nanomedicine · 2026Review
- How Effective are Exosomes in Overcoming Blood-Labyrinth Barrier in Sensorineural Hearing Loss? A Comprehensive Review of the Literature.International journal of nanomedicine · 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
- Mesenchymal stem cell-derived exosomes ameliorate gentamicin-induced vestibular hair cell injury by regulating the SNARE pathway and enhancing autophagy.Stem cell research & therapy · 2025Article
- Extracellular vesicle-mediated gene editing for the treatment of nonsyndromic progressive hearing loss in adult mice.Science translational medicine · 2025Article
- Round window membrane extracellular vesicles facilitate inner ear drug delivery.Journal of controlled release : official journal of the Controlled Release Society · 2025Article
- Efficient Scaling up EV-AAVs Production via Cellular Nanoporation for Familial Hypercholesterolaemia Therapy.Journal of extracellular vesicles · 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
- Extracellular vesicles for targeted drug delivery: advances in surface modification strategies and therapeutic applications.Journal of translational medicine · 2025Review
- IsMedComm · 2025Article
100 more citing papers are in PubMed but not listed here.
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Authors and funding
16 authors at 3 institutions in 2 countries.
Funding
Abstract
Adeno-associated virus (AAV) is a safe and effective vector for gene therapy for retinal disorders. Gene therapy for hearing disorders is not as advanced, in part because gene delivery to sensory hair cells of the inner ear is inefficient. Although AAV transduces the inner hair cells of the mouse cochlea, outer hair cells remain refractory to transduction. Here, we demonstrate that a vector, exosome-associated AAV (exo-AAV), is a potent carrier of transgenes to all inner ear hair cells. Exo-AAV1-GFP is more efficient than conventional AAV1-GFP, both in mouse cochlear explants in vitro and with direct cochlear injection in vivo. Exo-AAV shows no toxicity in vivo, as assayed by tests of auditory and vestibular function. Finally, exo-AAV1 gene therapy partially rescues hearing in a mouse model of hereditary deafness (lipoma HMGIC fusion partner-like 5/tetraspan membrane protein of hair cell stereocilia [Lhfpl5/Tmhs
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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.