Evidence map›Paper›PMID 41086995›Full record

ReviewMolecules and cells2025

Recent preclinical and clinical advances in gene therapy for hereditary hearing loss.

Seung Hyun Jang, Hyeong Gi Song, Jinsei Jung, Heon Yung Gee

Abstract readReview
In one paragraph

Review in Molecules and cells, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
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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

4 authors.

Seung Hyun JangDepartment of Pharmacology, Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul 03722, Republic of Korea; Won-Sang Lee Institute for Hearing Loss, Seoul 03722, Republic of Korea.
Hyeong Gi SongDepartment of Pharmacology, Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul 03722, Republic of Korea; Won-Sang Lee Institute for Hearing Loss, Seoul 03722, Republic of Korea.
Jinsei JungWon-Sang Lee Institute for Hearing Loss, Seoul 03722, Republic of Korea; Department of Otorhinolaryngology, Yonsei University College of Medicine, Seoul 03722, Republic of Korea.
Heon Yung GeeWon-Sang Lee Institute for Hearing Loss, Seoul 03722, Republic of Korea; Department of Otorhinolaryngology, Yonsei University College of Medicine, Seoul 03722, Republic of Korea; Woo Choo Lee Institute for Precision Drug Development, Seoul 03722, Republic of Korea. Electronic address: hygee@yuhs.ac.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hereditary hearing loss is a genetically heterogeneous condition that affects millions of people worldwide and has limited curative treatment options. Recent advancements in gene therapy have opened promising avenues for correcting the underlying genetic defects in the inner ear. This review summarizes the key developments in vector platforms, delivery strategies, target genes, preclinical models, and clinical trials relevant to both gene supplementation and gene editing approaches, as well as future directions. Adeno-associated virus vectors have emerged as the leading platform for inner ear gene transfer, owing to their safety and efficacy. Clinical programs, such as those targeting OTOF variants, are currently underway and are supported by robust preclinical data. Additionally, genome editing technologies, including CRISPR/Cas9-mediated nonhomologous end joining, base editing, and prime editing, offer variant-specific therapeutic potential. Despite these advances, challenges remain in expanding the therapeutic window, ensuring long-term safety, and establishing ethical and regulatory frameworks for their use.

Indexed as

Genetic TherapyHearing LossAnimalsClinical Trials as TopicCRISPR-Cas SystemsDependovirusGene EditingGenetic VectorsGene Transfer TechniquesHumansAdeno-associated virusGene editingGene supplementationHearing lossInner ear

Identifiers

PMID41086995
PMCPMC12605010

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.