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Cancer-specific trans-splicing ribozymes and their uses

机译:癌症特异的转拼核酶及其用途

摘要

PROBLEM TO BE SOLVED: To provide a cancer-specific trans-splicing ribozyme and its use. The present invention relates to a cancer-specific trans-splicing ribozyme and its use. The trans-splicing ribozyme according to the present invention does not act on normal tissues and is specifically expressed in cancer tissues, so that it is highly safe and has excellent expression efficiency at the post-transcriptional level, and is therefore usefully used in the treatment of cancer. obtain. Specifically, the recombinant vector of the present invention has a CMV promotor, SD / SA and WPRE, so that the expression efficiency of ribozyme is high, and miR-122T is possessed and the activity can be regulated by miR-122. , Excellent safety. Therefore, among liver cancers caused by HCV, miR-122 can be effectively used in the treatment of all liver cancers except for some liver cancers in which the expression level of miR-122 is higher in cancer tissues than in normal tissues. It can also be effectively used to treat other carcinomas that are not substantially expressed. [Selection diagram] Fig. 1
机译:要解决的问题:提供癌症特异性的转拼核酶及其用途。本发明涉及癌症特异性的转拼核酶及其用途。根据本发明的反式拼接核酶不作用于正常组织并且在癌症组织中特异性表达,因此它是高度安全的,并且在转录后水平上具有优异的表达效率,因此可用于治疗癌症。获得。具体地,本发明的重组载体具有CMV启动子,SD / SA和WPRE,因此核酶的表达效率高,并且具有miR-122T并且可以通过miR-122调节活性。 ,极好的安全性。因此,在由HCV引起的肝癌中,miR-122可以有效地用于所有肝癌的治疗,除了某些miR-122在癌症组织中的表达水平高于正常组织的肝癌。它也可以有效地用于治疗其他基本上未表达的癌。 [选择图]图1

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