Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model
Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model
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Publisher
United States: National Academy of Sciences
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Language
English
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Publisher
United States: National Academy of Sciences
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Contents
Autosomal recessive genetic forms (DFNB) account for most cases of profound congenital deafness. Adeno-associated virus (AAV)-based gene therapy is a promising therapeutic option, but is limited by a potentially short therapeutic window and the constrained packaging capacity of the vector. We focus here on the otoferlin gene underlying DFNB9, one o...
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Full title
Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6410774
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6410774
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ISSN
0027-8424
E-ISSN
1091-6490
DOI
10.1073/pnas.1817537116
