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公开(公告)号:EP3202909A1
公开(公告)日:2017-08-09
申请号:EP15847264.7
申请日:2015-07-23
IPC分类号: C12N15/85 , C12N9/22 , A61K31/713 , A61K38/43 , A61P35/00
CPC分类号: C12N15/113 , A61K31/713 , A61K38/00 , A61K38/43 , A61K48/0058 , A61K48/0066 , C07K14/4705 , C07K2319/01 , C07K2319/33 , C07K2319/61 , C12N9/22 , C12N15/1135 , C12N15/63 , C12N15/79 , C12N15/85 , C12N15/86 , C12N2310/113 , C12N2310/12 , C12N2310/124 , C12N2310/128 , C12N2330/51 , C12N2710/10343 , C12N2830/008 , C12N2830/42 , C12N2830/48
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.
摘要翻译: 本发明涉及重组载体,导入重组载体的转化细胞,由该重组载体表达的核酶,含有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; 和包含靶向癌症特异性基因的转拼核酶和连接于核酶的3'外显子的靶基因的核酶靶基因表达盒,其中连接剪接供体/剪接受体序列(SD / SA序列) 到核酶靶基因表达盒的5'末端,将土拨鼠肝炎病毒转录后调节元件(WPRE)连接到核酶靶基因表达盒的3'末端,并且将识别微小RNA-122a的核酸序列 (microRNA-122a,miR-122a)进一步连接到WPRE的3'末端。