{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,25]],"date-time":"2026-07-25T05:35:00Z","timestamp":1784957700847,"version":"3.55.0"},"reference-count":25,"publisher":"Wiley","issue":"29","license":[{"start":{"date-parts":[[2025,1,27]],"date-time":"2025-01-27T00:00:00Z","timestamp":1737936000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2025,1,27]],"date-time":"2025-01-27T00:00:00Z","timestamp":1737936000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100000055","name":"National Institute on Deafness and Other Communication Disorders","doi-asserted-by":"publisher","award":["R01DC015111"],"award-info":[{"award-number":["R01DC015111"]}],"id":[{"id":"10.13039\/100000055","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000055","name":"National Institute on Deafness and Other Communication Disorders","doi-asserted-by":"publisher","award":["R01DC008853"],"award-info":[{"award-number":["R01DC008853"]}],"id":[{"id":"10.13039\/100000055","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000055","name":"National Institute on Deafness and Other Communication Disorders","doi-asserted-by":"publisher","award":["R01DC018919"],"award-info":[{"award-number":["R01DC018919"]}],"id":[{"id":"10.13039\/100000055","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["advanced.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Advanced Science"],"published-print":{"date-parts":[[2025,8]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Usher syndrome type 1C (USH1C) is a genetic disorder caused by mutations in the USH1C gene, which encodes harmonin, a key component of the mechanoelectrical transduction complex in auditory and vestibular hair cells. USH1C leads to deafness and vestibular dysfunction in humans. An\n                    <jats:italic>Ush1c<\/jats:italic>\n                    knockout (KO) mouse model displaying these characteristic deficits is generated in our laboratory. To examine gene replacement therapy (GT) in this model, a synthetic adeno\u2010associated viral vector,\n                    <jats:italic>Anc80L65<\/jats:italic>\n                    , driving harmonin expression is administered, to the inner ears of\n                    <jats:italic>Ush1c<\/jats:italic>\n                    KO mice at postnatal day 2 (P2). Remarkably, this single treatment significantly improved auditory brainstem response (ABR) thresholds and balance motor function at 1 month post\u2010injection, with these effects persisting for up to 10 months. At 12 months post\u2010treatment, the vestibular function is assessed using the vestibular\u2010ocular reflexes (VOR) and single vestibular afferent recordings. The GT treatment significantly restored both the canal and otolith VORs and increased vestibular afferent spontaneous firing rates and responses to head rotation and translation. These findings provide the first evidence of long\u2010lasting restoration of both the auditory and vestibular functions by GT in a novel mouse model of Usher syndrome, highlighting the potential of GT for treating deficits associated with USH1C.\n                  <\/jats:p>","DOI":"10.1002\/advs.202410063","type":"journal-article","created":{"date-parts":[[2025,1,28]],"date-time":"2025-01-28T00:40:56Z","timestamp":1738024856000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Long\u2010Lasting Auditory and Vestibular Recovery Following Gene Replacement Therapy in a Novel Usher Syndrome Type 1c Mouse Model"],"prefix":"10.1002","volume":"12","author":[{"given":"Weinan","family":"Du","sequence":"first","affiliation":[{"name":"Department of Otolaryngology Case Western Reserve University  Cleveland OH 44106 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jun","family":"Huang","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology\u2010Head and Neck Surgery University of Mississippi Medical Center  Jackson MS 39216 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aizhen","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology Case Western Reserve University  Cleveland OH 44106 USA"},{"name":"Department of Otolaryngology\u2010Head and Neck Surgery Shandong Provincial ENT Hospital Shandong University  Jinan Shandong 250022 China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fangfang","family":"Zhao","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology Case Western Reserve University  Cleveland OH 44106 USA"},{"name":"Hearing and Speech Rehabilitation Institute Binzhou Medical University  Yantai 264000 China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tianwen","family":"Chen","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology\u2010Head and Neck Surgery University of Mississippi Medical Center  Jackson MS 39216 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Quinn M.","family":"McDermott","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology Case Western Reserve University  Cleveland OH 44106 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tony","family":"Zheng","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology Case Western Reserve University  Cleveland OH 44106 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Haibo","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology\u2010Head and Neck Surgery Shandong Provincial ENT Hospital Shandong University  Jinan Shandong 250022 China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rongli","family":"Zhang","sequence":"additional","affiliation":[{"name":"Case Cardiovascular Research Institute Case Western Reserve University School of Medicine  Cleveland OH 44106 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaolin","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology Binzhou Medical University Hospital  Binzhou 256603 China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerome","family":"Allison","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology\u2010Head and Neck Surgery University of Mississippi Medical Center  Jackson MS 39216 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hong","family":"Zhu","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology\u2010Head and Neck Surgery University of Mississippi Medical Center  Jackson MS 39216 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wu","family":"Zhou","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology\u2010Head and Neck Surgery University of Mississippi Medical Center  Jackson MS 39216 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5827-4106","authenticated-orcid":false,"given":"Qing Yin","family":"Zheng","sequence":"additional","affiliation":[{"name":"Department of Otolaryngology Case Western Reserve University  Cleveland OH 44106 USA"},{"name":"Department of Otolaryngology Binzhou Medical University Hospital  Binzhou 256603 China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,1,27]]},"reference":[{"key":"e_1_2_9_1_1","doi-asserted-by":"publisher","DOI":"10.2471\/BLT.21.285643"},{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajhg.2018.08.006"},{"key":"e_1_2_9_2_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00439-023-02559-9"},{"key":"e_1_2_9_2_3","unstructured":"c)W. 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