首页> 外国专利> ADENOVIRAL VECTOR WITH REPLICATION-DEPENDENT TRANSGENE EXPRESSION

ADENOVIRAL VECTOR WITH REPLICATION-DEPENDENT TRANSGENE EXPRESSION

机译:具有复制依赖性转基因表达的腺病毒载体

摘要

The invention provides an adenoviral vector comprising (a) at least a portion of an adenoviral genome comprising a major late transcription unit containing a terminal exon, wherein the terminal exon comprises a 5' splice acceptor DNA sequence element and a 3' polyadenylation signal sequence, and (b) a non-native nucleic acid sequence encoding a protein that does not contribute to the adenoviral vector entry into a host cell, wherein the non-native nucleic acid sequence is positioned within the terminal exon, such that the nonnative nucleic acid sequence is selectively expressed in cells within which the adenoviral vector can replicate. The invention further provides an adenoviral vector composition and a method for treating or preventing a pathologic state in a mammal, comprising administering to the mammal the adenoviral vector composition of the invention in an amount sufficient to treat or prevent the pathologic state in the mammal.
机译:本发明提供了一种腺病毒载体,其包含(a)腺病毒基因组的至少一部分,其包含含有末端外显子的主要晚期转录单元,其中所述末端外显子包含5'剪接受体DNA序列元件和3'聚腺苷酸化信号序列, (b)编码不有助于腺病毒载体进入宿主细胞的蛋白质的非天然核酸序列,其中所述非天然核酸序列位于末端外显子内,使得所述非天然核酸序列在腺病毒载体可以在其中复制的细胞中选择性地表达Aβ1。本发明进一步提供了用于治疗或预防哺乳动物病理状态的腺病毒载体组合物和方法,包括以足以治疗或预防哺乳动物病理状态的量向哺乳动物给药本发明的腺病毒载体组合物。

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