RNA TARGETING COMPOUNDS AND METHODS FOR MAKING AND USING SAME

摘要

Disclosed are RNA targeting compounds having the formula : wherein j is a n integer from 1 to 100; each i is the same or different and is zero or an i nteger from 1 to 100; each Z1 represents the same or different linking moiet y; each R1 is the same or different and represents an alkyl group or an aryl group; each Q1 represents the same or different RNA binding ligand; Q2 is a n alkyl group; Q3 is a halogen, an alkyl group, an aryl group, or an amine. Also disclosed are RNA targeting compounds that include a polymer backbone a nd two or more pendant RNA binding ligands that are bound to the polymer bac kbone. Methods for using the subject RNA targeting compounds to treat myoton ic dystrophy and other diseases are also disclosed, as are compounds that ca n be used to prepare the subject RNA targeting compounds.