ArticlePloS one2012
Inner ear gene transfection in neonatal mice using adeno-associated viral vector: a comparison of two approaches.
Article in PloS one, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 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
28 citing papers in PubMed, 1 synthesis or guideline pooled it, 39 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
- The predawn dilemma in adeno-associated virus-based gene therapies for hereditary deafness.American journal of stem cells · 2025Review
- A base editor for the long-term restoration of auditory function in mice with recessive profound deafness.Nature biomedical engineering · 2025Article
- Gene Therapy for Inherited Hearing Loss: Updates and Remaining Challenges.Audiology research · 2023Review
- Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Towards the Clinical Application of Gene Therapy for Genetic Inner Ear Diseases.Journal of clinical medicine · 2023Review
- Genetic insights, disease mechanisms, and biological therapeutics for Waardenburg syndrome.Gene therapy · 2022Review
- Optimized AAV Vectors for TMC1 Gene Therapy in a Humanized Mouse Model of DFNB7/11.Biomolecules · 2022Article
- Advances in genome editing for genetic hearing loss.Advanced drug delivery reviews · 2021Review
- Neurotrophin gene therapy to promote survival of spiral ganglion neurons after deafness.Hearing research · 2020Review
- Gene therapy development in hearing research in China.Gene therapy · 2020Review
- rAAV-Mediated Cochlear Gene Therapy: Prospects and Challenges for Clinical Application.Journal of clinical medicine · 2020Review
- Self-complementarity in adeno-associated virus enhances transduction and gene expression in mouse cochlear tissues.PloS one · 2020Article
- Genetic Therapies for Hearing Loss: Accomplishments and Remaining Challenges.Neuroscience letters · 2019Review
- Gene therapy for hearing loss.Human molecular genetics · 2019Review
- Cochlear Gene Therapy.Cold Spring Harbor perspectives in medicine · 2019Review
- AAV-Mediated Neurotrophin Gene Therapy Promotes Improved Survival of Cochlear Spiral Ganglion Neurons in Neonatally Deafened Cats: Comparison of AAV2-hBDNF and AAV5-hGDNF.Journal of the Association for Research in Otolaryngology : JARO · 2019Article
- Anatomical and Surgical Evaluation of the Common Marmoset as an Animal Model in Hearing Research.Frontiers in neuroanatomy · 2019Article
- Virally Mediated Overexpression of Glial-Derived Neurotrophic Factor Elicits Age- and Dose-Dependent Neuronal Toxicity and Hearing Loss.Human gene therapy · 2019Article
- Canalostomy As a Surgical Approach to Local Drug Delivery into the Inner Ears of Adult and Neonatal Mice.Journal of visualized experiments : JoVE · 2018Article
Corrections and comments
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Authors and funding
3 authors at 2 institutions in 2 countries.
Funding
Abstract
Local gene transfection is a promising technique for the prevention and/or correction of inner ear diseases, particularly those resulting from genetic defects. Adeno-associated virus (AAV) is an ideal viral vector for inner ear gene transfection because of its safety, stability, long-lasting expression, and its high tropism for many different cell types. Recently, a new generation of AAV vectors with a tyrosine mutation (mut-AAV) has demonstrated significant improvement in transfection efficiency. A method for inner ear gene transfection via the intact round window membrane (RWM) has been developed in our laboratory. This method has not been tested in neonatal mice, an important species for the study of inherited hearing loss. Following a preliminary study to optimize the experimental protocol in order to reduce mortality, the present study investigated inner ear gene transfection in mice at postnatal day 7. We compared transfection efficiency, the safety of the scala tympani injection via RWM puncture, and the trans-RWM diffusion following partial digestion with an enzyme technique. The results revealed that approximately 47% of inner hair cells (IHCs) and 17% of outer hair cells (OHCs) were transfected via the trans-RWM approach. Transfection efficiency via RWM puncture (58% and 19% for IHCs and OHCs, respectively) was slightly higher, but the difference was not significant.
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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.