Evidence map›Paper›PMID 41495521›Full record

ReviewEuropean archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery2026

Stem cell-based and gene editing strategies in the treatment of congenital and acquired sensorineural hearing loss: a narrative review.

Alireza Moradi, Seied-Reza Seied-Mohammad Doulabi, Ashkan Sherafat Vaziri, Silke Helbig, Mehran Baghi

Abstract readReview
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In one paragraph

Review in European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

5 authors.

Alireza MoradiDepartment of Otolaryngology, SINA Clinic, Otto Wels Straße 5, Obertshausen, 63179, Germany.ORCID http://orcid.org/0000-0002-4692-3463
Seied-Reza Seied-Mohammad DoulabiDepartment of Otolaryngology, SINA Clinic, Otto Wels Straße 5, Obertshausen, 63179, Germany.ORCID http://orcid.org/0000-0002-8321-8034
Ashkan Sherafat VaziriDepartment of Otolaryngology, SINA Clinic, Otto Wels Straße 5, Obertshausen, 63179, Germany.ORCID http://orcid.org/0000-0001-5770-0030
Silke HelbigDepartment of Otorhinolaryngology, University Hospital Frankfurt, Frankfurt/M, Germany.ORCID http://orcid.org/0009-0004-0679-0610
Mehran BaghiDepartment of Otolaryngology, SINA Clinic, Otto Wels Straße 5, Obertshausen, 63179, Germany. m.baghi@mvz-sina.de.ORCID http://orcid.org/0009-0003-3040-6642

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundSensorineural hearing loss (SNHL) represents the most common sensory deficit in humans and is increasingly prevalent with aging. Despite diverse etiologies, irreversible degeneration of cochlear hair cells and neurons remains a final pathway for auditory decline.

objectiveThis narrative review synthesizes current preclinical and early clinical evidence on gene- and stem cell-based strategies for congenital and acquired SNHL, highlighting translational progress, delivery innovations, and persisting limitations.

methodsA structured literature and registry search (PubMed, Embase, Scopus, Web of Science, ClinicalTrials.gov, EudraCT) was conducted using Boolean combinations of disease-, gene-, vector-, and delivery-related terms. From 140 initial records, 46 eligible studies were included after full-text screening. Data were qualitatively analyzed across two streams-human clinical (gene therapy) and preclinical/experimental (gene editing, RNA-based modulation, stem-cell regeneration).

resultsTwo independent first-in-human AAV-mediated OTOF replacement trials in children with DFNB9 demonstrated partial restoration of hearing, establishing the first clinical proof-of-concept for cochlear gene therapy. Preclinical studies confirm durable auditory and vestibular rescue using dual-AAV systems, while CRISPR-based editing, antisense oligonucleotides (ASOs), and RNA interference (RNAi) approaches show mechanistic feasibility. Induced pluripotent stem cells (iPSCs) and inner-ear organoids replicate key developmental pathways, supporting modeling and future cellular repair. Delivery precision via the round window, oval window, and microneedle systems remains the major translational bottleneck.

conclusionsGene- and cell-based approaches are transforming auditory regenerative medicine. Among them, AAV-mediated OTOF replacement has reached clinical validation, whereas CRISPR, ASO/RNAi, and iPSC-derived regeneration continue at advanced preclinical stages. Future success will depend on refining delivery, ensuring long-term safety, and harmonizing ethical and regulatory oversight.

Indexed as

Gene EditingGenetic TherapyHearing Loss, SensorineuralStem Cell TransplantationAnimalsHumansAntisense oligonucleotidesCochlear regenerationCRISPRGene therapyInduced pluripotent stem cellsOTOFRNA interferenceRound windowSensorineural hearing lossTranslational otology.

Identifiers

PMID41495521

What OpenQuestion holds

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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.