PHOSPHORODIAMIDATE MORPHOLINO OLIGOMERS (PMOS) AND THEIR USE IN SUPPRESSION OF MUTANT HUNTINGTIN EXPRESSION AND ATTENUATION OF NEUROTOXICITY
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United States of America Patent
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Issued Date -
Jan 21, 2016
app pub date -
Jul 6, 2015
filing date -
Jul 11, 2014
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Abstract
The present invention provides antisense phosphorodiamidate morpholino oligomers which are useful for the suppression or inhibition of the HTT gene involved in Huntington's disease. The oligomers can selectively suppress mutant forms of the HTT protein while allowing the normal protein to be expressed in sufficient quantity to retain its function in the cell. Methods for treatment of Huntington's disease are also provided.
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