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Cancer-specific trans-splicing ribozymes and uses thereof

机译:癌症特异的转拼核酶及其用途

摘要

The present invention relates to a recombination vector, a transformation cell into which the recombinant vector is introduced, a ribozyme expressed from the recombination vector, a prophylactic or therapeutic composition for liver cancer comprising the recombination vector and the ribozyme, and a therapeutic method for liver cancer using the composition, said recombination vector comprising: a tissue-specific promoter; and a ribozyme-target gene expression cassette comprising a trans-splicing ribozyme targeting a cancer-specific gene and a target gene connected to the 3' exon of the ribozyme, wherein a splicing donor/splicing acceptor sequence (SD/SA sequence) is connected to the 5' end of the ribozyme-target gene expression cassette, woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) is connected to the 3' end of the ribozyme-target gene expression cassette, and a nucleic acid sequence recognizing a micro RNA-122a (microRNA-122a, miR-122a) is further connected to the 3' end of the WPRE.
机译:本发明涉及重组载体,导入了重组载体的转化细胞,由重组载体表达的核酶,包含该重组载体和核酶的肝癌的预防或治疗组合物以及肝的治疗方法使用所述组合物的癌症,所述重组载体包含:组织特异性启动子;和核糖酶靶基因表达盒,其包含靶向癌症特异性基因的转拼核酶和连接至核酶3'外显子的靶基因,其中剪接供体/剪接受体序列(SD / SA序列)被连接在核酶靶基因表达盒的5'端,土拨鼠肝炎病毒转录后调控元件(WPRE)连接到核酶靶基因表达盒的3'端,并带有识别微小RNA-122a的核酸序列(microRNA-122a,miR-122a)进一步连接到WPRE的3'端。

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