A CODON OPTIMIZED OTOFERLIN AAV DUAL VECTOR GENE THERAPY
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机译:密码子优化的OTOFERLIN AAV双载体基因治疗
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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 herein are compositions of AAV particles comprising one or more polynucleotides encoding Otoferlin are codon optimized for expression in human cells. Such methods and compositions may be useful for treatment of diseases and disorders such as Deafness, Autosomal Recessive 9 (DFNB9). Also provided are kits comprising such compositions.
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