Evidence map›Paper›PMID 40538303›Full record

ReviewAdvanced science (Weinheim, Baden-Wurttemberg, Germany)2025

Exploring AAV-Mediated Gene Therapy for Inner Ear Diseases: from Preclinical Success to Clinical Potential.

Fan Wu, Wuhui He, Yun Xiao, Qiong Wu, Qing Zhang, Tiesong Zhang, Lei Xu, Xinyi Pang

Abstract readReview
In one paragraph

Review in Advanced science (Weinheim, Baden-Wurttemberg, Germany), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 2 pooled it
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

6 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
  2. Pooled it
  3. Article
  4. Review
  5. Review
  6. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Fan WuDepartment of Pathology and Laboratory Medicine, The Medical University of South Carolina, Walton Research Building, Room 403-E, Charleston, SC, 29425, USA.
Wuhui HeDepartment of Otolaryngology, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, Guangdong, 510000, China.
Yun XiaoDepartment of Otolaryngology-Head and Neck Surgery, Shandong Provincial ENT Hospital, Shandong University, Jinan, Shandong, 250000, China.
Qiong WuDepartment of Otorhinolaryngology-Head and Neck Surgery, Xinhua Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China/Ear Institute, Shanghai Jiaotong University School of Medicine, Shanghai, China/Shanghai Key Laboratory of Translational Medicine on Ear and Nose Diseases, Shanghai, 200001, China.
Qing ZhangDepartment of Otorhinolaryngology-Head and Neck Surgery, Xinhua Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China/Ear Institute, Shanghai Jiaotong University School of Medicine, Shanghai, China/Shanghai Key Laboratory of Translational Medicine on Ear and Nose Diseases, Shanghai, 200001, China.
Tiesong ZhangYunnan Key Laboratory of Children's Major Disease Research, Yunnan Province Clinical Research Center for Children's Health and Disease, Yunnan Institute of Pediatrics, Kunming Key Laboratory of Children Infection and Immunity, Kunming Children's Hospital, Kunming, Yunnan, 650228, China.
Lei XuDepartment of Otolaryngology-Head and Neck Surgery, Shandong Provincial ENT Hospital, Shandong University, Jinan, Shandong, 250022, China.
Xinyi PangCollege of Food Science and Engineering, Nanjing University of Finance and Economics, Nanjing, 210023, China.ORCID https://orcid.org/0000-0001-9628-1377

Funding

Medicine-Science and Engineering Interdisciplinary Foundation of Shanghai Hospital Development Center SHDC2022CRD013National Natural Science Foundation of China 32200484National Natural Science Foundation of China 82060192National Natural Science Foundation of China 82071053National Natural Science Foundation of China 82171137National Natural Science Foundation of China 82201277Natural Science Foundation of Jiangsu Province BK20241904School of Medicine, Shanghai Jiao Tong University 23XHCR05ASpecial Funds for High-level Healthy Talents of Yunnan Province-Medical Leaders L-2019002Taishan Scholar Foundation of Shandong Province tsqn202211357Yunnan International Science and Technology Co-Laboratory of Allergic Disease 202103AF140008Yunnan Key Laboratory of Children's Major Disease Research 202005AG070073Yunnan Province Clinical Research Center for Children's Health and Disease 202102AA100064
6 · The paper itself

Abstract

Hearing loss imposes a significant global health burden and often results from genetic factors and various external influences, such as noise exposure and the use of ototoxic drugs. Recent advancements in adeno-associated virus (AAV)-mediated gene therapy offer promising new avenues for the treatment of inner ear diseases. Clinical trials of AAV-mediated gene therapies show impressive preliminary results, although further research is needed to evaluate the safety and long-term effects of these therapies. Preclinical AAV-mediated gene therapy is notable for its high transduction efficiency and safety. In this article, the development of AAV-mediated gene therapies is reviewed for inner ear diseases, categorizing these therapies by their strategies for treating hereditary hearing loss, including gene replacement and cluster regularly interspaced short palindromic repeat-based methods. AAV-mediated hair cell regeneration therapy is also reviewed for acquired hearing loss, as well as methods to prevent acquired hearing loss. In this article, it is hoped to provide a comprehensive overview of recent progress in AAV-mediated gene therapy and its future potential, thereby highlighting the importance of this therapy for researchers and clinicians in the field.

Indexed as

DependovirusGenetic TherapyHearing LossLabyrinth DiseasesAnimalsGenetic VectorsHumansAAVsacquired hearing losscochleargene therapiesgenetic hearing lossinner earssensory hair cells

Identifiers

PMID40538303
PMCPMC12412547

What OpenQuestion holds

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Registered trials

None linked

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.