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GENE THERAPIES FOR USHER SYNDROME (USH2A)
GENE THERAPIES FOR USHER SYNDROME (USH2A)
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机译:用户综合症(USH2A)的基因治疗
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摘要
Aspects of the disclosure relate to compositions and methods useful for delivering minigenes to a subject. Accordingly, the disclosure is based, in part, on isolated nucleic acids and gene therapy vectors, such as viral (e.g., rAAV) vectors, comprising one or more gene fragments encoding a therapeutic gene product, such as a protein or peptide (e.g., a minigene). In some embodiments, the disclosure relates to gene therapy vectors encoding a USH2A protein (e.g., the gene product of USH2A gene) or a portion thereof. In some embodiments, compositions described by the disclosure are useful for treating diseases associated with mutations in the USH2A gene, for example Usher Syndrome.
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