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http://purl.uniprot.org/citations/34910522http://www.w3.org/1999/02/22-rdf-syntax-ns#typehttp://purl.uniprot.org/core/Journal_Citation
http://purl.uniprot.org/citations/34910522http://www.w3.org/2000/01/rdf-schema#comment"Hearing loss affects an estimated 466 million people worldwide, with a substantial fraction due to genetic causes. Approximately 16% of genetic hearing loss is caused by pathogenic mutations in STRC, a gene that encodes the protein stereocilin. To develop gene therapy strategies for patients with STRC hearing loss, we generated a mouse model with a targeted deletion in the Strc gene. We devised a novel dual-vector approach to circumvent the size limitation of AAV vectors and drive expression of full-length STRC protein. To target outer hair cells, which are difficult to transduce, we used synthetic AAV9-PHP.B vectors for efficient dual-vector transduction. We report robust recovery of exogenous STRC expression in outer hair cells of Strc-deficient mice, recovery of hair bundle morphology, substantially improved cochlear amplification, and enhanced auditory sensitivity. The data raise the prospect that our strategy could benefit ~2.3 million patients worldwide affected by STRC mutations."xsd:string
http://purl.uniprot.org/citations/34910522http://purl.org/dc/terms/identifier"doi:10.1126/sciadv.abi7629"xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/author"French C."xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/author"Holt J.R."xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/author"Shearer A.E."xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/author"Rockowitz S."xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/author"Nist-Lund C."xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/author"Shubina-Oleinik O."xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/date"2021"xsd:gYear
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/name"Sci Adv"xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/pages"eabi7629"xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/title"Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16 hearing loss."xsd:string
http://purl.uniprot.org/citations/34910522http://purl.uniprot.org/core/volume"7"xsd:string
http://purl.uniprot.org/citations/34910522http://www.w3.org/2004/02/skos/core#exactMatchhttp://purl.uniprot.org/pubmed/34910522
http://purl.uniprot.org/citations/34910522http://xmlns.com/foaf/0.1/primaryTopicOfhttps://pubmed.ncbi.nlm.nih.gov/34910522
http://purl.uniprot.org/uniprot/#_F6UNX5-mappedCitation-34910522http://www.w3.org/1999/02/22-rdf-syntax-ns#objecthttp://purl.uniprot.org/citations/34910522
http://purl.uniprot.org/uniprot/#_Q3UMD0-mappedCitation-34910522http://www.w3.org/1999/02/22-rdf-syntax-ns#objecthttp://purl.uniprot.org/citations/34910522
http://purl.uniprot.org/uniprot/#_Q8VIM6-mappedCitation-34910522http://www.w3.org/1999/02/22-rdf-syntax-ns#objecthttp://purl.uniprot.org/citations/34910522
http://purl.uniprot.org/uniprot/Q8VIM6http://purl.uniprot.org/core/mappedCitationhttp://purl.uniprot.org/citations/34910522
http://purl.uniprot.org/uniprot/Q3UMD0http://purl.uniprot.org/core/mappedCitationhttp://purl.uniprot.org/citations/34910522
http://purl.uniprot.org/uniprot/F6UNX5http://purl.uniprot.org/core/mappedCitationhttp://purl.uniprot.org/citations/34910522