http://purl.uniprot.org/citations/34910522 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
http://purl.uniprot.org/citations/34910522 | http://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/34910522 | http://purl.org/dc/terms/identifier | "doi:10.1126/sciadv.abi7629"xsd:string |
http://purl.uniprot.org/citations/34910522 | http://purl.uniprot.org/core/author | "French C."xsd:string |
http://purl.uniprot.org/citations/34910522 | http://purl.uniprot.org/core/author | "Holt J.R."xsd:string |
http://purl.uniprot.org/citations/34910522 | http://purl.uniprot.org/core/author | "Shearer A.E."xsd:string |
http://purl.uniprot.org/citations/34910522 | http://purl.uniprot.org/core/author | "Rockowitz S."xsd:string |
http://purl.uniprot.org/citations/34910522 | http://purl.uniprot.org/core/author | "Nist-Lund C."xsd:string |
http://purl.uniprot.org/citations/34910522 | http://purl.uniprot.org/core/author | "Shubina-Oleinik O."xsd:string |
http://purl.uniprot.org/citations/34910522 | http://purl.uniprot.org/core/date | "2021"xsd:gYear |
http://purl.uniprot.org/citations/34910522 | http://purl.uniprot.org/core/name | "Sci Adv"xsd:string |
http://purl.uniprot.org/citations/34910522 | http://purl.uniprot.org/core/pages | "eabi7629"xsd:string |
http://purl.uniprot.org/citations/34910522 | http://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/34910522 | http://purl.uniprot.org/core/volume | "7"xsd:string |
http://purl.uniprot.org/citations/34910522 | http://www.w3.org/2004/02/skos/core#exactMatch | http://purl.uniprot.org/pubmed/34910522 |
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