A CODON OPTIMIZED OTOFERLIN AAV DUAL VECTOR GENE THERAPY
作者： Dyka Frank , Hauswirth William
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摘要: Provided herein are methods and compositions for expressing Otoferlin, e.g., utilizing adeno-associated viral (AAV) particles. Further provided of AAV particles comprising one or more polynucleotides encoding Otoferlin codon optimized expression in human cells. Such may be useful treatment diseases disorders such as Deafness, Autosomal Recessive 9 (DFNB9). Also kits compositions.
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