{"doi":"10.3390/ijms22083910","title":"Usher Syndrome in the Inner Ear: Etiologies and Advances in Gene Therapy","abstract":"Hearing loss is the most common sensory disorder with ~466 million people worldwide affected, representing about 5% of the population. A substantial portion of hearing loss is genetic. Hearing loss can either be non-syndromic, if hearing loss is the only clinical manifestation, or syndromic, if the hearing loss is accompanied by a collage of other clinical manifestations. Usher syndrome is a syndromic form of genetic hearing loss that is accompanied by impaired vision associated with retinitis pigmentosa and, in many cases, vestibular dysfunction. It is the most common cause of deaf-blindness. Currently cochlear implantation or hearing aids are the only treatments for Usher-related hearing loss. However, gene therapy has shown promise in treating Usher-related retinitis pigmentosa. Here we review how the etiologies of Usher-related hearing loss make it a good candidate for gene therapy and discuss how various forms of gene therapy could be applied to Usher-related hearing loss.","journal":"International Journal of Molecular Sciences","year":2021,"id":170793,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":27,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9564,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2021-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":438096,"name":"Brett Colbert","orcid":"0000-0002-9668-0674","position":1,"is_corresponding":false},{"id":357646,"name":"Pei-Ciao Tang","orcid":null,"position":2,"is_corresponding":false},{"id":396779,"name":"Byron L. Lam","orcid":"0000-0002-1233-2366","position":3,"is_corresponding":false},{"id":404905,"name":"Jun Yang","orcid":"0000-0001-6978-9968","position":4,"is_corresponding":false},{"id":514510,"name":"Susan H. Blanton","orcid":"0000-0002-5433-3439","position":5,"is_corresponding":false},{"id":392129,"name":"Derek M. Dykxhoorn","orcid":"0000-0002-4998-9943","position":6,"is_corresponding":false},{"id":513722,"name":"Xue Zhong Liu","orcid":"0000-0002-4758-6470","position":7,"is_corresponding":false},{"id":703859,"name":"Evan de Joya","orcid":"0000-0002-5259-1039","position":0,"is_corresponding":true}],"reference_count":133,"raw_metadata":null,"created_at":"2026-07-18T23:46:24.040760Z","pmid":"33920085","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}