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USE OF ADENO-ASSOCIATED VIRAL VECTORS TO CORRECT GENE DEFECTS/ EXPRESS PROTEINS IN HAIR CELLS AND SUPPORTING CELLS IN THE INNER EAR
USE OF ADENO-ASSOCIATED VIRAL VECTORS TO CORRECT GENE DEFECTS/ EXPRESS PROTEINS IN HAIR CELLS AND SUPPORTING CELLS IN THE INNER EAR
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机译:使用腺瘤相关的病毒载体纠正毛细胞中的基因缺陷/表达蛋白质,并在内耳中支持细胞
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摘要
Provided herein are compositions that include at least two different nucleic acid vectors that, when introduced into a primate cell, the at least two different vectors undergo concatamerization or homologous recombination with each other, thereby forming a recombined nucleic acid that encodes a full-length target protein (e.g., a supporting cell target protein or a hair cell target protein). Also provided are compositions that include a single AAV vector that, when introduced into a primate cell, a nucleic acid encoding a full-length target protein (e.g., a supporting cell target protein or a hair cell target protein) is generated at the locus of the supporting cell target gene, and the primate expresses the target protein (e.g., a supporting cell target protein or a hair cell target protein).
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