ArticleMolecular therapy : the journal of the American Society of Gene Therapy2013
Mouse otocyst transuterine gene transfer restores hearing in mice with connexin 30 deletion-associated hearing loss.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.
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Who cites it
41 citing papers in PubMed, 77 citations in OpenAlex.
- IsMedComm · 2025Article
- Effect of Wendan decoction granules on preventing endolymphatic hydrops and protecting vestibular function in Guinea pigs.Hereditas · 2025Article
- Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations.Mammalian genome : official journal of the International Mammalian Genome Society · 2025Review
- Mitochonic acid 5 mitigates age-related hearing loss progression by targeting defective 2-methylthiolation in mitochondrial transfer RNAs.Frontiers in cellular neuroscience · 2025Article
- AAV-mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Review
- Gene therapy for hearing loss: challenges and the promise of cellular plasticity and epigenetic modulation.Frontiers in neurology · 2024Article
- Article
- Mitochondrial alterations in the cochlea of Cdk5rap1-knockout mice with age-related hearing loss.FEBS open bio · 2023Article
- Arginine-Rich Cell-Penetrating Peptide-Mediated Transduction of Mouse Nasal Cells with FOXP3 Protein Alleviates Allergic Rhinitis.Pharmaceutics · 2023Article
- Recent advances and future challenges in gene therapy for hearing loss.Royal Society open science · 2023Review
- Advances in gene therapy hold promise for treating hereditary hearing loss.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- Towards the Clinical Application of Gene Therapy for Genetic Inner Ear Diseases.Journal of clinical medicine · 2023Review
- The Genomics of Auditory Function and Disease.Annual review of genomics and human genetics · 2022Review
- Virally Mediated Connexin 26 Expression in Postnatal Scala Media Significantly and Transiently Preserves Hearing in Connexin 30 Null Mice.Frontiers in cell and developmental biology · 2022Article
- Dynamic Spatiotemporal Expression Changes in Connexins of the Developing Primate's Cochlea.Genes · 2021Article
- Cdk5 regulatory subunit-associated protein 1 knockout mice show hearing loss phenotypically similar to age-related hearing loss.Molecular brain · 2021Article
- Gene Therapy to the Retina and the Cochlea.Frontiers in neuroscience · 2021Review
- Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65.Molecular therapy. Methods & clinical development · 2020Article
- Fetal gene therapy and pharmacotherapy to treat congenital hearing loss and vestibular dysfunction.Hearing research · 2020Review
- Inner Ear Gene Therapies Take Off: Current Promises and Future Challenges.Journal of clinical medicine · 2020Review
Corrections and comments
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Although numerous causative genes for hereditary hearing loss have been identified, there are no fundamental treatments for this condition. Herein, we describe a novel potential treatment for genetic hearing loss. Because mutations or deletions in the connexin (Cx) genes are common causes of profound congenital hearing loss in both humans and mice, we investigated whether gene supplementation therapy using the wild-type Cx gene could cure hearing loss. We first generated inner ear-specific connexin 30 (Cx30)-deficient mice via the transuterine transfer of Cx30-targeted short hairpin RNA (shRNA-Cx30) into otocysts. The inner ear-specific Cx30-deficient mice mimicked homozygous Cx30-deficient mice both histologically and physiologically. Subsequently, we cotransfected the shRNA-Cx30 and the wild-type Cx30 gene. The cotransfected mice exhibited Cx30 expression in the cochleae and displayed normal auditory functions. Next, we performed the transuterine transfer of the wild-type Cx30 gene into the otocysts of homozygous Cx30-deficient mice, thereby rescuing the lack of Cx30 expression in the cochleae and restoring auditory functioning. These results demonstrate that supplementation therapy with wild-type genes can restore postnatal auditory functioning. Moreover, this is the first report to show that Cx-related genetic hearing loss is treatable by in vivo gene therapy.
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