ArticleAdvanced science (Weinheim, Baden-Wurttemberg, Germany)2024
Preclinical Efficacy And Safety Evaluation of AAV-OTOF in DFNB9 Mouse Model And Nonhuman Primate.
Erratum issuedOpen access · goldAbstract read
In one paragraph
Article in Advanced science (Weinheim, Baden-Wurttemberg, Germany), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 47 papers.
0numbers the graph read from it
0cells of the map it votes in
47citing papers in PubMed
11.2field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
47 citing papers in PubMed, 55 citations in OpenAlex.
- AAV gene therapy for autosomal recessive deafness 9: a single-arm trial.Nature medicine · 2025Trial
- Parental experience of whole genome sequencing for children with sensorineural hearing loss.International journal of qualitative studies on health and well-being · 2026Article
- Review
- Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness.Molecular therapy. Advances · 2026Article
- Rescue of stereocilia architecture and hearing function by AAV-CIB2 and AAV-CIB3 in a mouse model of recessive deafness DFNB48.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Age outweighs route for AAV-PHP.eB transgene distribution via inner ear delivery.Drug delivery and translational research · 2026Article
- Using Electrospinning Technique for Rapid Preparation of BDNF-Releasing Electrode Array of Cochlear Implant: An In Vitro Study.ACS omega · 2026Article
- Long-term restoration of auditory function in a DFNA2 mouse model by adenine base editing.EMBO molecular medicine · 2026Article
- Human promoter-driven AAV tools enable precision gene therapy targeting cochlear hair cells.Cell reports. Medicine · 2026Article
- Postnatal Slc26a4 gene therapy improves hearing and structural integrity in a hereditary hearing loss model.The Journal of clinical investigation · 2026Article
- Pathogenic mechanism of the PLS1 gene variant in hearing loss and functional validation in a zebrafish model.Scientific reports · 2026Article
- Generation of marmoset monkeys with a non-mosaic disruption of the OTOF gene as a model of human deafness.Nature communications · 2026Article
- Superenhancer-mediated ferroptosis in age-related hearing loss: cochlear epigenomics.Cellular and molecular life sciences : CMLS · 2026Article
- Cidea Targeting Protects Cochlear Hair Cells and Hearing Function From Drug- and Noise-Induced Damage.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- The Current Situation and Development of Endoscopic Ear Surgery in China.World journal of otorhinolaryngology - head and neck surgery · 2026Review
- Bioactive hydrogels for the reconstruction of sensorineural hearing loss.Bioactive materials · 2026Review
- Pou4f3 Deficiency Obstructs the Subtype Differentiation of Vestibular Hair Cells.Neuroscience bulletin · 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
- Engineered Inner Ear Drug Delivery Systems for Hearing Loss Treatment.Research (Washington, D.C.) · 2026Review
- Transcriptomic Profiling Reveals Divergent Immune Responses to AAV1 and AAV-ie in Mice Inner Ear.International journal of medical sciences · 2026Article
Corrections and comments
- Erratum issued
Authors and funding
18 authors at 5 institutions in 1 country.
Jieyu QiState Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, 210096, China.
Liyan ZhangState Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, 210096, China.
Fangzhi TanState Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, 210096, China.
Yang ZhangDepartment of Neurology, Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing, 210008, China.
Yinyi ZhouState Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, 210096, China.
Ziyu ZhangState Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, 210096, China.
Hongyang WangSenior Department of Otolaryngology-Head & Neck Surgery, the Sixth Medical Center of Chinese PLA General Hospital, Beijing, 100048, China.
Chaorong YuOtovia Therapeutics Inc., Suzhou, 215101, China.
Lulu JiangOtovia Therapeutics Inc., Suzhou, 215101, China.
Jiancheng LiuOtovia Therapeutics Inc., Suzhou, 215101, China.
Tian ChenState Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, 210096, China.
Lianqiu WuOtovia Therapeutics Inc., Suzhou, 215101, China.
Shanzhong ZhangOtovia Therapeutics Inc., Suzhou, 215101, China.
Sijie SunOtovia Therapeutics Inc., Suzhou, 215101, China.
Shan SunENT Institute and Otorhinolaryngology, Department of Affiliated Eye and ENT Hospital, Key Laboratory of Hearing Medicine of NHFPC, Fudan University, Shanghai, 200031, China.
Ling LuDepartment of Otolaryngology-Head and Neck Surgery, Jiangsu Provincial Key Medical Discipline (Laboratory), Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing, 210008, China.
Qiuju WangSenior Department of Otolaryngology-Head & Neck Surgery, the Sixth Medical Center of Chinese PLA General Hospital, Beijing, 100048, China.
Renjie ChaiState Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, 210096, China.ORCID 0000-0002-3885-543X
Zhongda Hospital Southeast University · CNBeijing Institute of Technology · CNChinese PLA General Hospital · CNFosun Pharma (China) · CNNanjing Drum Tower Hospital · CN
Funding
2022 Open Project Fund of Guangdong Academy of Medical Sciences YKY-KF202201China National Postdoctoral Program for Innovative Talents BX20200082China Postdoctoral Science Foundation 2020M681468Fundamental Research Funds for the Central UniversitiesJiangsu Postdoctoral Research Funding Program 2021K156BNanjing Medical Science and Technology Development Project YKK19072National Key Research and Development Program of China 2020YFA0112503National Key Research and Development Program of China 2020YFA0113600National Key Research and Development Program of China 2021YFA1101300National Key Research and Development Program of China 2021YFA1101800National Natural Science Foundation of China 82000984National Natural Science Foundation of China 82030029National Natural Science Foundation of China 82071059National Natural Science Foundation of China 82330033National Natural Science Foundation of China 82371161National Natural Science Foundation of China 82371162National Natural Science Foundation of China 92149304Science and Technology Department of Sichuan Province 2021YFS0371Shenzhen Science and Technology Program JCYJ20190814093401920Shenzhen Science and Technology Program JCYJ20210324125608022STI2030-Major Projects 2022ZD0205400
Abstract
OTOF mutations are the principal causes of auditory neuropathy. There are reports on Otof-related gene therapy in mice, but there is no preclinical research on the drug evaluations. Here, Anc80L65 and the mouse hair cell-specific Myo15 promoter (mMyo15) are used to selectively and effectively deliver human OTOF to hair cells in mice and nonhuman primates to evaluate the efficacy and safety of OTOF gene therapy drugs. A new dual-AAV-OTOF-hybrid strategy to transfer full-length OTOF is generated, which can stably restore hearing in adult OTOF
Indexed as
Hearing LossHearing Loss, SensorineuralAdultAnimalsHumansMembrane ProteinsMicePrimatesMembrane ProteinsOTOF protein, humangene therapynonhuman primateOTOFpreclinical research
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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.