Article
Recovery kinetics of dual AAV-mediated human otoferlin expression
17 Jun 2024
Abstract excerpt
Deafness-causing deficiencies in otoferlin ( OTOF ) have been addressed preclinically using dual adeno-associated virus (AAV)-based approaches. However, timing of transduction, recombination of mRNA, and protein expression with dual hybrid AAV methods methods have not previously been characterized. Here, we have established an ex vivo assay to determine the kinetics of dual-AAV mediated expression of OTOF in hair...
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