The Uab Research Foundation

Ribozyme Pharmaceuticals, Incorporated

Valorisation-recherche, Limited Partnership

University of Montreal

Ribozyme Pharmacueticals, Inc.

Pascal Chartrand

Nassim Usman

Robert J Cedergren

Stephen C Harvey

Emmanuelle Querido

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.

Inhibitors of polynucleotide repeat-associated RNA foci and uses thereof

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:

Catalytic DNA

Growing community of inventors

Montreal, Canada

Pascal Chartrand