Uses of pentamidine and related compounds
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United States of America Patent
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May 7, 2013
Grant Date -
Dec 23, 2010
app pub date -
Feb 20, 2009
filing date -
Feb 21, 2008
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Expired
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Abstract
Methods are provided herein for treatment of myotonic dystrophy and other toxic RNA diseases in a subject. In some examples, the method comprises administration of a compound that binds a nucleotide repeat expansion in a ribonucleic acid molecule, thereby treating the disease. In additional examples, the method comprises administration of a compound that disrupts binding of muscleblind-like proteins to an RNA nucleotide repeat expansion. Compounds for use in the disclosed method include pentamidine or heptamidine or derivatives thereof. Representative compounds are described herein.
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