Evidence map›Paper›PMID 40236500›Full record

ArticleMolecular therapy. Methods & clinical development2025

AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear.

Yasuko Ishibashi, Jianliang Zhu, Gwladys Gernoux, Yunkai Yu, Michelle J Suh, Kevin Isgrig, Mhamed Grati, Rafal Olszewski, Michael Hoa, Cao Liang and 3 more

Abstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed, 1 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

4 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Recent Progress in Mechanism-Based Therapies forInternational journal of molecular sciences · 2026
    Review
  4. Article
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

13 authors.

Yasuko IshibashiInner Ear Gene Therapy Program, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD 20892, USA.
Jianliang ZhuInner Ear Gene Therapy Program, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD 20892, USA.
Gwladys GernouxNantes Université, CHU de Nantes, INSERM, TaRGeT - Translational Research in Gene Therapy, UMR 1089, F-44200 Nantes, France.
Yunkai YuOMICS Technology Facility, Genetics Branch, The Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Michelle J SuhInner Ear Gene Therapy Program, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD 20892, USA.
Kevin IsgrigInner Ear Gene Therapy Program, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD 20892, USA.
Mhamed GratiInner Ear Gene Therapy Program, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD 20892, USA.
Rafal OlszewskiAuditory Development and Restoration Program, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD 20892, USA.
Michael HoaAuditory Development and Restoration Program, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD 20892, USA.
Cao LiangOMICS Technology Facility, Genetics Branch, The Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Thomas B FriedmanLaboratory of Molecular Genetics, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD 20892, USA.
Oumeya AdjaliNantes Université, CHU de Nantes, INSERM, TaRGeT - Translational Research in Gene Therapy, UMR 1089, F-44200 Nantes, France.
Wade W ChienInner Ear Gene Therapy Program, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, MD 20892, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hearing loss is a common disability affecting the world's population. Currently, its treatment options are limited. Adeno-associated virus (AAV)-mediated inner ear gene therapy has shown great promise as a treatment for hereditary hearing loss. However, the host immune responses to AAV-mediated gene therapy in the mammalian inner ear is not well understood. In this study, two serotypes of AAV vectors were injected individually into the mouse inner ear to evaluate the host innate and adaptive immune responses up to 1 month after inner ear gene delivery. Our results suggest that the host innate and adaptive immune responses to AAV-mediated inner ear gene delivery are limited and mild, which is favorable for its clinical translation.

Indexed as

adaptive immune responseadeno-associated virusddPCREPLSpotgene therapyinnate immune responseinner earneutralizing antibodypro-inflammatory cytokines

Identifiers

PMID40236500
PMCPMC11999604

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

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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.