Cancer-specific trans-splicing ribozymes and use thereof
AssigneesRznomics Inc., Dong-A University Research Foundation for Industry-Academy Cooperation, Industry-Academic Cooperation Foundation, Dankook University
InventorsSeong-Wook Lee, Chang Ho Lee, Seung Ryul I Han, Ji Hyun Kim, Eun Yi Cho, Jin Sook Jeong, Mi Ha Ju
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Abstract
A cancer-specific trans-splicing ribozyme and a use thereof are disclosed. The trans-splicing ribozyme does not act on normal tissue, but is specifically expressed in cancer tissue. Therefore, it is very safe and has excellent expression efficiency at the post-transcription level, and thus can be effectively used in treatment of cancer.
Claims21
A nucleic acid construct, comprising, in this order from 5′ end to 3′ end:
- (i) a cytomegalovirus (CMV) promoter;
- (ii) a ribozyme-desired gene expression cassette, which includes (a) a sequence encoding a trans-splicing ribozyme targeting a cancer-specific gene and (b) a target gene connected to a 3′ exon of the ribozyme-encoding sequence,
- wherein the nucleic acid construct has a splicing donor/splicing acceptor sequence (SD/SA sequence) connected to the 5′ end of the ribozyme-desired gene expression cassette, and a Woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) connected to the 3′ end of the ribozyme-desired gene expression cassette;
- and
- (iii) a nucleic acid sequence recognizing microRNA-122 (miR-122), which is connected to the 3′ end of the WPRE.
14-17. (canceled).
Dependencies are inferred from the claim text.