Johns Hopkins University

Ionis Pharmaceuticals, Inc.

Constantin Van Outryve D'Ydewalle

C Frank Bennett

Frank Rigo

Charlotte J Sumner

Certain embodiments are directed to methods and compounds for modulating expression of SMN. In certain embodiments at least two compounds are used: a first compound for inhibiting SMN-NAT and increasing expression of SMN, and a second compound for modulating the splicing of SMN. Such methods and compounds are useful for increasing expression exon 7 containing SMN mRNA in cells and animals.

Combinations for the modulation of SMN expression

Certain embodiments are directed to methods and compounds for inhibiting SMN-NAT, the natural antisense transcript of SMN. Such methods and compounds are useful for increasing expression of SMN in cells and animals.

Growing community of inventors

Baltimore, MD, United States of America

Constantin Van Outryve D'Ydewalle