ArticleSignal transduction and targeted therapy2022
AAV-ie-K558R mediated cochlear gene therapy and hair cell regeneration.
Article in Signal transduction and targeted therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 papers.
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Who cites it
46 citing papers in PubMed, 66 citations in OpenAlex.
- Recent advancements in improving cross-species applicability of bioengineered AAV capsids.Gene therapy · 2026Review
- Functionalized Biomimetic Scaffolds for Human-Derived Auditory Neural Circuit Construction.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- The cochlear morphometry compendium: High-resolution synchrotron measurements and normative reference values.Journal of anatomy · 2026Article
- Capsid-engineered AAV vector overcomes a key intracellular barrier and efficiently transduces spiral ganglion neurons in adult mice.Molecular therapy. Advances · 2026Article
- Gene supplementation with precise transgene expression rescues hearing loss in a mouse model with an Mpzl2 East Asian founder variant.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Evolutionary constraints impose multiple checkpoints that block mammalian sensory hair cell regeneration.Frontiers in cell and developmental biology · 2026Review
- M/G-Ratio-Tuned Calcium Alginate Hydrogel Microcarrier for Controlled Delivery of Adeno-Associated Virus for Gene Therapy.Biotechnology and bioengineering · 2025Article
- Implication of GPRASP2 in the Proliferation and Hair Cell-Forming of Cochlear Supporting Cells.Cell proliferation · 2025Article
- ShH10 viral vector for safe, efficient, and selective transduction of inner ear supporting cells.Developmental biology · 2025Article
- Pvalb8, a Type of Oncomodulin, Regulates Neuromast Development and Auditory Function in Zebrafish.Cells · 2025Article
- Gene therapy restores auditory function and rescues damaged inner hair cells in an aged Vglut3 knockout mouse model.Gene therapy · 2025Article
- Rapid cochlear gene therapy in adult deaf mice:Molecular therapy. Methods & clinical development · 2025Article
- Exploring AAV-Mediated Gene Therapy for Inner Ear Diseases: from Preclinical Success to Clinical Potential.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- IsMedComm · 2025Article
- Viral-Mediated Connexin 26 Expression Combined with Dexamethasone Rescues Hearing in a Conditional Gjb2 Null Mice Model.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Single Administration of AAV-mAtp6v1b2 Gene Therapy Rescues Hearing and Vestibular Disorders Caused by Atp6v1b2-Induced Lysosomal Dysfunction in Hair Cells.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Presbycusis: Pathology, Signal Pathways, and Therapeutic Strategy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- The Reconstruction of Peripheral Auditory Circuit: Recent Advances and Future Challenges.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations.Mammalian genome : official journal of the International Mammalian Genome Society · 2025Review
- Therapeutic restoration of miR-96 prevents hearing loss in mice through modulation of noise-induced and genetic pathways.iScience · 2025Article
Corrections and comments
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Authors and funding
15 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The cochlea consists of multiple types of cells, including hair cells, supporting cells and spiral ganglion neurons, and is responsible for converting mechanical forces into electric signals that enable hearing. Genetic and environmental factors can result in dysfunctions of cochlear and auditory systems. In recent years, gene therapy has emerged as a promising treatment in animal deafness models. One major challenge of the gene therapy for deafness is to effectively deliver genes to specific cells of cochleae. Here, we screened and identified an AAV-ie mutant, AAV-ie-K558R, that transduces hair cells and supporting cells in the cochleae of neonatal mice with high efficiency. AAV-ie-K558R is a safe vector with no obvious deficits in the hearing system. We found that AAV-ie-K558R can partially restore the hearing loss in Prestin KO mice and, importantly, deliver Atoh1 into cochlear supporting cells to generate hair cell-like cells. Our results demonstrate the clinical potential of AAV-ie-K558R for treating the hearing loss caused by hair cell death.
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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.