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USE OF AAV INTEGRATION EFFICIENCY ELEMENT FOR MEDIATING SITE-SPECIFIC INTEGRATION OF A TRANSCRIPTION UNIT
USE OF AAV INTEGRATION EFFICIENCY ELEMENT FOR MEDIATING SITE-SPECIFIC INTEGRATION OF A TRANSCRIPTION UNIT
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机译:AAV集成效率元素在调解转录单位特定站点集成中的使用
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摘要
The invention provides an expression construct comprising a nucleic acid sequence encoding an adeno-associated virus integration efficiency element (AAV IEE), wherein the expression construct is substantially devoid of AAV inverted terminal repeats (AAV ITRs). Such an expression construct site-specifically integrates into a host cell chromosome when provided to a host cell in conjunction with an AAV Rep protein. The invention also provides a method of integrating a nucleic acid sequence of interest into a host cell chromosome through use of such an expression construct, as well as a method of prophylactically or therapeutically treating a mammal for a pathologic state comprising administering to a mammal such an expression construct comprising a nucleic acid sequence encoding a therapeutic factor.
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