Evidence map›Paper›PMID 36769694›Full record

ReviewJournal of clinical medicine2023

Towards the Clinical Application of Gene Therapy for Genetic Inner Ear Diseases.

Ghizlene Lahlou, Charlotte Calvet, Marie Giorgi, Marie-José Lecomte, Saaid Safieddine

Abstract readReview
In one paragraph

Review in Journal of clinical medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing 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

17 citing papers in PubMed.

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  14. Human cochlear diffusion from the cerebrospinal fluid space with gadolinium contrast.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Article
  15. Article
  16. 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

5 authors.

Ghizlene LahlouInstitut Pasteur/Institut de l'Audition, Technologie et Thérapie Génique de la Surdité, Sorbonne Université, INSERM, Sorbonne Université, 75012 Paris, France.ORCID 0000-0002-1067-045X
Charlotte CalvetInstitut Pasteur/Institut de l'Audition, Technologie et Thérapie Génique de la Surdité, Sorbonne Université, INSERM, Sorbonne Université, 75012 Paris, France.ORCID 0000-0002-6163-9807
Marie GiorgiInstitut Pasteur/Institut de l'Audition, Technologie et Thérapie Génique de la Surdité, Sorbonne Université, INSERM, Sorbonne Université, 75012 Paris, France.
Marie-José LecomteInstitut Pasteur/Institut de l'Audition, Technologie et Thérapie Génique de la Surdité, Sorbonne Université, INSERM, Sorbonne Université, 75012 Paris, France.
Saaid SafieddineInstitut Pasteur/Institut de l'Audition, Technologie et Thérapie Génique de la Surdité, Sorbonne Université, INSERM, Sorbonne Université, 75012 Paris, France.

Funding

Agence Nationale de la Recherche ANR-18-RHUS-0007Fondation pour l'Audition FPA IDA08
6 · The paper itself

Abstract

Hearing loss, the most common human sensory defect worldwide, is a major public health problem. About 70% of congenital forms and 25% of adult-onset forms of deafness are of genetic origin. In total, 136 deafness genes have already been identified and there are thought to be several hundred more awaiting identification. However, there is currently no cure for sensorineural deafness. In recent years, translational research studies have shown gene therapy to be effective against inherited inner ear diseases, and the application of this technology to humans is now within reach. We provide here a comprehensive and practical overview of current advances in gene therapy for inherited deafness, with and without an associated vestibular defect. We focus on the different gene therapy approaches, considering their prospects, including the viral vector used, and the delivery route. We also discuss the clinical application of the various strategies, their strengths, weaknesses, and the challenges to be overcome.

Indexed as

AAVgene therapyhearing impairmentvestibular defect

Identifiers

PMID36769694
PMCPMC9918244

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.