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ADENO-ASSOCIATED VIRUS WITH INVERTED TERMINAL REPEAT SEQUENCES AS PROMOTER FOR THE IN VIVO TRANSFER OF A FUNCTIONAL CFTR GENE
ADENO-ASSOCIATED VIRUS WITH INVERTED TERMINAL REPEAT SEQUENCES AS PROMOTER FOR THE IN VIVO TRANSFER OF A FUNCTIONAL CFTR GENE
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机译:具有倒转末端重复序列的腺伴随病毒,作为功能性CFTR基因体内转移的启动子
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摘要
Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.
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