RNA TARGETING COMPOUNDS AND METHODS FOR MAKING AND USING SAME

摘要

Disclosed are RNA targeting compounds having the formula : wherein j is an integer from 1 to 100; each i is the same or different and is zero or an integer from 1 to 100; each Z 1 represents the same or different linking moiety; each R 1 is the same or different and represents an alkyl group or an aryl group; each Q 1 represents the same or different RNA binding ligand; Q 2 is an alkyl group; Q 3 is a halogen, an alkyl group, an aryl group, or an amine. Also disclosed are RNA targeting compounds that include a polymer backbone and two or more pendant RNA binding ligands that are bound to the polymer backbone. Methods for using the subject RNA targeting compounds to treat myotonic dystrophy and other diseases are also disclosed, as are compounds that can be used to prepare the subject RNA targeting compounds.