Invention Application
- Patent Title: TRANSGENIC ANIMAL MODEL FOR SPLICEOSOME-MEDIATED RNA TRANS-SPLICING
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Application No.: PCT/US2003/013161Application Date: 2003-04-30
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Publication No.: WO2003093414A3Publication Date: 2003-11-13
- Inventor: PUTTARAJU, Madaiah , MITCHEL, Lloyd , ENGELHARDT, John, F. , BLANCO, Mariano, Garcia , LIU, Xiaoming
- Applicant: INTRONN, INC. , PUTTARAJU, Madaiah , MITCHEL, Lloyd , ENGELHARDT, John, F. , BLANCO, Mariano, Garcia , LIU, Xiaoming
- Applicant Address: 9700 Great Seneca Highway, Rocksville, MD 20850 US
- Assignee: INTRONN, INC.,PUTTARAJU, Madaiah,MITCHEL, Lloyd,ENGELHARDT, John, F.,BLANCO, Mariano, Garcia,LIU, Xiaoming
- Current Assignee: INTRONN, INC.,PUTTARAJU, Madaiah,MITCHEL, Lloyd,ENGELHARDT, John, F.,BLANCO, Mariano, Garcia,LIU, Xiaoming
- Current Assignee Address: 9700 Great Seneca Highway, Rocksville, MD 20850 US
- Agency: SEIDE, Rochelle, K.
- Priority: US10/136,723 20020430
- Main IPC: G01N33/00
- IPC: G01N33/00
Abstract:
The present invention relates to development of an animal model system for in vivo testing of spliceosome-mediated RNA trans -splicing reactions. The present invention provides transgenic animals, and methods for generating such animals, that have been genetically engineered to expresses a target precursor messenger RNA molecule (target pre-mRNA) that serves as a substrate for a trans -splicing reaction. Specifically, the transgenic animals contain at least one transgene capable of expressing a target pre-mRNA molecule. The invention provides methods, based on utilization of the transgenic animals, for assessing the specificity and efficiency of a pre- trans -splicing molecule (PTM) designed to interact with a target pre-mRNA and mediate a trans -splicing reaction resulting in the generation of a novel chimeric RNA molecule. The present invention further relates to the transgenic expression of PTM molecules in animals to determine gene function, i.e , functional genetics. The present invention is based on the successful generation of a transgenic animal expressing a target pre-mRNA and, moreover, the use of that animal to detect accurate in vivo trans -splicing reactions in the presence of a PTM.
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