Abstract: Compounds which inhibit the formation and/or accumulation of RNA foci, such as those due to polynucleotide repeats (e.g., trinucleotide repeats) are described herein. Also described herein are uses of such compounds, such as for the inhibition of the formation and accumulation such RNA foci, as well as for the treatment of polynucleotide repeat disorders (e.g., trinucleotide repeat disorders), such as myotonic dystrophy (e.g., DM1). Such compounds include compounds of formula 1, 1a, 1b, 2, 2a and (3) described herein.

Abstract: Compounds which inhibit the formation and/or accumulation of RNA foci, such as those due to polynucleotide repeats (e.g., trinucleotide repeats) are described herein. Also described herein are uses of such compounds, such as for the inhibition of the formation and accumulation such RNA foci, as well as for the treatment of polynucleotide repeat disorders (e.g., trinucleotide repeat disorders), such as myotonic dystrophy (e.g., DM1). Such compounds include compounds of formula 1, 1a, 1b, 2, 2a and (3) described herein.

Abstract: Nucleic acid able to cause specific cleavage of a bond between two ribonucleotides in an RNA-containing molecule. The RNA-containing molecule has the structure:5'-XnUHZCUGANGAGYm-3'wherein each X and Y is independently any nucleotide base; n and m are independently between 5 and 40; H is U, A or C; Z is a hairpin loop, having between 6 and 60 bases, and each U, C, G and A is a uracil, cytosine, guanosine, or adenosine-containing ribonucleotide, respectively, and N is any ribonucleotide. The nucleic acid has the structure:3'-X'nM.sub.o Y'm-5'wherein each X' and Y' are complementary nucleotide bases to each corresponding X and Y, and M.sub.o is a series of nucleotide bases active to cause the cleavage, and wherein M.sub.o contains no ribonucleotides.

Abstract: Nucleic acid able to cause specific cleavage of a bond between two ribonucleotides in an RNA-containing molecule. The RNA-containing molecule has the structure:5'-XnUHZCUGANGAGYm-3'wherein each X and Y is independently any nucleotide base; n and m are independently between 5 and 40; H is U, A or C; Z is a hairpin loop, having between 6 and 60 bases, and each U, C, G and A is a uracil, cytosine, guanosine, or adenosine-containing ribonucleotide, respectively, and N is any ribonucleotide. The nucleic acid has the structure:3'-X'nM.sub.0 Y'm-5'wherein each X' and Y' are complementary nucleotide bases to each corresponding X and Y, and M.sub.0 is a series of nucleotide bases active to cause the cleavage, and wherein M.sub.0 contains no ribonucleotides.