TAILED MIRTRON EFFECTORS FOR RNAI-MEDIATED GENE SILENCING
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United States of America Patent
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Jan 5, 2017
app pub date -
Oct 17, 2014
filing date -
Dec 3, 2013
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Abstract
The present invention relates to an isolated mirtron capable of binding a target nucleic acid, wherein the isolated mirtron comprises: a) a stem loop structure comprising a 5′ splice site, b) a tail sequence comprising a 3′ splice site and a polypyrimidine-comprising sequence, and c) a branch point sequence. Further, the present invention relates to vectors and pharmaceutical compositions comprising such mirtrons as well as methods of treating a disease comprising administering such pharmaceutical compositions.
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