{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T06:35:53Z","timestamp":1791959753720,"version":"4.3.4"},"reference-count":140,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2023,1,29]],"date-time":"2023-01-29T00:00:00Z","timestamp":1674950400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Fondation pour l\u2019Audition","award":["FPA IDA08"],"award-info":[{"award-number":["FPA IDA08"]}]},{"name":"Fondation pour l\u2019Audition","award":["ANR-18-RHUS-0007"],"award-info":[{"award-number":["ANR-18-RHUS-0007"]}]},{"DOI":"10.13039\/501100001665","name":"RHU AUDINNOVE","doi-asserted-by":"publisher","award":["FPA IDA08"],"award-info":[{"award-number":["FPA IDA08"]}],"id":[{"id":"10.13039\/501100001665","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001665","name":"RHU AUDINNOVE","doi-asserted-by":"publisher","award":["ANR-18-RHUS-0007"],"award-info":[{"award-number":["ANR-18-RHUS-0007"]}],"id":[{"id":"10.13039\/501100001665","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>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.<\/jats:p>","DOI":"10.3390\/jcm12031046","type":"journal-article","created":{"date-parts":[[2023,1,30]],"date-time":"2023-01-30T01:33:37Z","timestamp":1675042417000},"page":"1046","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":39,"title":["Towards the Clinical Application of Gene Therapy for Genetic Inner Ear Diseases"],"prefix":"10.3390","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1067-045X","authenticated-orcid":false,"given":"Ghizlene","family":"Lahlou","sequence":"first","affiliation":[{"name":"Institut Pasteur\/Institut de l\u2019Audition, Technologie et Th\u00e9rapie G\u00e9nique de la Surdit\u00e9, Sorbonne Universit\u00e9, INSERM, Sorbonne Universit\u00e9, 75012 Paris, France"},{"name":"D\u00e9partement d\u2019Oto-Rhino-Laryngologie, Unit\u00e9 Fonctionnelle Implants Auditifs, Groupe Hospitalo-Universitaire Piti\u00e9-Salp\u00eatri\u00e8re, APHP Sorbonne Universit\u00e9, 75013 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6163-9807","authenticated-orcid":false,"given":"Charlotte","family":"Calvet","sequence":"additional","affiliation":[{"name":"Institut Pasteur\/Institut de l\u2019Audition, Technologie et Th\u00e9rapie G\u00e9nique de la Surdit\u00e9, Sorbonne Universit\u00e9, INSERM, Sorbonne Universit\u00e9, 75012 Paris, France"},{"name":"Zurich Integrative Rodent Physiology (ZIRP), University of Zurich, CH-8057 Zurich, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie","family":"Giorgi","sequence":"additional","affiliation":[{"name":"Institut Pasteur\/Institut de l\u2019Audition, Technologie et Th\u00e9rapie G\u00e9nique de la Surdit\u00e9, Sorbonne Universit\u00e9, INSERM, Sorbonne Universit\u00e9, 75012 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie-Jos\u00e9","family":"Lecomte","sequence":"additional","affiliation":[{"name":"Institut Pasteur\/Institut de l\u2019Audition, Technologie et Th\u00e9rapie G\u00e9nique de la Surdit\u00e9, Sorbonne Universit\u00e9, INSERM, Sorbonne Universit\u00e9, 75012 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saaid","family":"Safieddine","sequence":"additional","affiliation":[{"name":"Institut Pasteur\/Institut de l\u2019Audition, Technologie et Th\u00e9rapie G\u00e9nique de la Surdit\u00e9, Sorbonne Universit\u00e9, INSERM, Sorbonne Universit\u00e9, 75012 Paris, France"},{"name":"Centre National de la Recherche Scientifique, 75016 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,1,29]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"879","DOI":"10.1016\/S0140-6736(05)71047-3","article-title":"Sensorineural Hearing Loss in Children","volume":"365","author":"Smith","year":"2005","journal-title":"Lancet"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"996","DOI":"10.1016\/S0140-6736(21)00516-X","article-title":"Hearing Loss Prevalence and Years Lived with Disability, 1990\u20132019: Findings from the Global Burden of Disease Study 2019","volume":"397","author":"Haile","year":"2021","journal-title":"Lancet"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1016\/j.anorl.2015.08.026","article-title":"Schooling of Hearing-Impaired Children and Benefit of Early Diagnosis","volume":"132","author":"Boukhris","year":"2015","journal-title":"Eur. 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