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adeno assozierter virus with inverted terminal repeat sequences as a promoter for the in vivo transfer of a functional cftr gene
adeno assozierter virus with inverted terminal repeat sequences as a promoter for the in vivo transfer of a functional cftr gene
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机译:具有反向末端重复序列的腺assozierter病毒作为体内cftr基因转移的启动子
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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. IMAGE
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