Spliceosome mediated RNA trans-splicing
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United States of America Patent
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Issued Date -
Oct 28, 2004
app pub date -
Mar 20, 2002
filing date -
Mar 20, 2002
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Abandoned
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Abstract
The invention provides molecules and methods for in vivo production of a trans-spliced molecule in selected cells. Pre-trans-splicing molecules of the invention are substrates for a trans-splicing reaction between the pre-trans-splicing molecules and a pre-mRNA which is uniquely expressed in the specific target cells. The in vivo trans-splicing reaction provides a novel mRNA which is functional as mRNA or encodes a protein to be expressed in the target cells. The mRNA expression product is a therapeutic protein, a toxin which causes killing of the specific cells, or a novel protein not normally present in such cells. The invention further provides genetically engineered PTMs for the identification of exon/intron boundaries of pre-mRNA molecules using an exon tagging method. The PTMs of the invention can also be designed to produce chimeric RNA encoding peptide affinity purification tags which can be used to purify and identify proteins expressed in a specific cell type.
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