Evidence map›Paper›PMID 41508981›Full record

ArticleClinical and translational medicine2026

Dual AAV gene therapy achieves recovery of hearing and auditory processing in a DFNB16 mouse model.

Sepideh Iranfar, Sophie Bagur, Chloé Felgerolle, Maxence Cornille, Najate Benamer, Hélène Le Ribeuz, Marie Giorgi, Sara Jamali, Amel Saoudi, Marie-José Lecomte and 3 more

Abstract read
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In one paragraph

Article in Clinical and translational medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–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

2 citing papers in PubMed.

  1. Article
  2. 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.

Sepideh IranfarUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0001-5615-3290
Sophie BagurUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0002-4375-1838
Chloé FelgerolleUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0001-8824-375X
Maxence CornilleUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0002-8369-1104
Najate BenamerUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.
Hélène Le RibeuzUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0002-6579-6076
Marie GiorgiUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0002-5505-7165
Sara JamaliUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0003-4933-3167
Amel SaoudiUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.
Marie-José LecomteUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0001-5097-6798
Vincent MichelInstitut Pasteur, INSERM, Pathogenesis of Vascular Infections Unit, Paris, France.ORCID 0000-0002-7025-4343
Brice BathellierUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0001-9211-1960
Saaid SafieddineUniversité Paris Cité, Institut Pasteur, AP-HP, INSERM, CNRS, Fondation Pour l'Audition, Institut de l'Audition, IHU reConnect, Paris, France.ORCID 0000-0002-6159-0572

Funding

Agence Nationale de la Recherche 'EARGENCURE' ANR-17-CE18-0027Agence Nationale de la Recherche 'HEAR AGAIN' ANR 24-CE17-1002Agence Nationale de la Recherche 'RHU AUDINNOVE' ANR-18-RHUS-0007Agence Nationale de la Recherche 'TIME-TO-EAR' ANR-23-CE17-0029European Research Council ERCCoG770841492DEEPENFondation pour la Recherche Médicale SPF202005011970Fondation pour l'Audition 'FPAIDA08','RD-2023-1','FPAIDA02','APA2016-03'France 2030 program ANR-23-IAHU-0003
6 · The paper itself

Abstract

backgroundDFNB16, the second most common genetic cause of hearing loss, is caused by mutations of the STRC gene encoding stereocilin, a protein essential for the effective functioning of outer hair cells (OHCs) as cochlear amplifiers. Strc

methodsUsing Strc

resultsDual-AAV-mediated Strc gene delivery restored stereocilin expression, OHC bundle architecture and their attachment to the tectorial membrane, leading to the recovery of cochlear amplification and hearing to near normal thresholds, as confirmed by distortion product otoacoustic emission (DPOAE) and auditory brainstem response measurements. Behavioural assessment showed that treated Strc

conclusionThese findings provide the first proof-of-principle that peripheral gene therapy can restore OHC function, cochlear amplification and central auditory perception in a DFNB16 model. KEY POINTS: Dual AAV-mediated gene delivery restored peripheral hearing in a DFNB16 preclinical mouse model. The same treatment also restored central auditory processing. AAV-mediated gene therapy represents a promising curative strategy for DFNB16. These results reinforce the translational potential for treating human genetic deafness.

Indexed as

DependovirusGenetic TherapyHearingAnimalsAuditory AcuityDisease Models, AnimalEvoked Potentials, Auditory, Brain StemGene Therapy AgentsMiceAAVDFNB16gene therapyhearingsound processingstereocilin

Identifiers

PMID41508981
PMCPMC12784207

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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.