ArticleGene therapy2020
Adeno-associated virus vector enables safe and efficient Cas9 activation in neonatal and adult Cas9 knockin murine cochleae.
Article in Gene therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 19 citations in OpenAlex.
- Cochlear nucleus spatial transcriptomes of normal and hearing loss mice reveal a critical role of Spp1 in bushy cells.Cell research · 2026Article
- 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
- AAVR Expression is Essential for AAV Vector Transduction in Sensory Hair Cells.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Targeted spiral ganglion neuron degeneration in parvalbumin-Cre neonatal mice.Molecular therapy. Methods & clinical development · 2025Article
- Gene therapy breakthroughs in ALS: a beacon of hope for 20% of ALS patients.Translational neurodegeneration · 2025Review
- Current AAV-mediated gene therapy in sensorineural hearing loss.Fundamental research · 2025Review
- AAV-mediated Gene Therapy for Hereditary Deafness: Progress and Perspectives.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Review
- Gene therapy: an emerging therapy for hair cells regeneration in the cochlea.Frontiers in neuroscience · 2023Review
- Approaches and Vectors for Efficient Cochlear Gene Transfer in Adult Mouse Models.Biomolecules · 2022Article
- Adeno-Associated Viral Vectors as Versatile Tools for Neurological Disorders: Focus on Delivery Routes and Therapeutic Perspectives.Biomedicines · 2022Review
- Inner Ear Drug Delivery for Sensorineural Hearing Loss: Current Challenges and Opportunities.Frontiers in neuroscience · 2022Review
- Article
- Gene Therapy to the Retina and the Cochlea.Frontiers in neuroscience · 2021Review
- Spotlight on gene therapy in China.Gene therapy · 2020Article
- Inner Ear Gene Delivery: Vectors and Routes.Hearing, balance and communication · 2020Article
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adeno-associated virus (AAV)-mediated gene delivery systems have been shown to be effective tools for gene manipulation in the inner ear. For example, hair cells (HCs) and multiple other cell types can be transduced by the local injection of AAVs into the inner ear. However, application of the AAV-mediated CRISPR/Cas9 gene-editing approach to the inner ear in adult mice has not yet been studied. Based on our previous work, we investigated several AAV serotypes in neonatal and adult mice in parallel, and found that AAV8 had the top efficiency to transduce inner HCs. We then tested the ability of Cre-expressing AAV8 to activate Cas9 in floxed-Cas9 knockin mice, and observed significant Cas9 activation in the inner ear of both neonatal and adult animals. Neither the AAV8 virus itself nor the surgical procedures used to deliver it-cochleostomy for neonatal mice and canalostomy for adult mice-caused any damage to HCs or impaired normal hearing. Our studies indicate that the local injection of AAV8-Cre can induce Cas9 activation to perform safe and efficient gene editing in the inner ear, expanding the repertoire of gene-editing tools for regulating gene expression in the inner ear as a part of efforts to rescue genetic hearing loss, initiate regeneration of HCs, or develop gene therapy techniques.
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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.