Chimeric RNA molecules generated by trans-splicing
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United States of America Patent
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Jan 11, 2000
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N/A
app pub date -
Dec 13, 1996
filing date -
Dec 15, 1995
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Abstract
The molecules and methods of the present invention provide a means for in vivo production of a therapeutic molecule in a selected subset of cells. The pre-therapeutic molecules of the invention are substrates for a trans-splicing reaction between the pre-therapeutic molecules and a pre-mRNA which is uniquely expressed in the specific target cells. The in vivo trans-splicing reaction provides an active therapeutic RNA which is functional as RNA or encodes a protein to be expressed in the target cells. The expression product of the mRNA is a protein of therapeutic value to the cell or a toxin which causes killing of the specific cells.
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