Modulation of SMN expression
AssigneesIonis Pharmaceuticals, Inc., The Johns Hopkins University
InventorsFrank Rigo, C. Frank Bennett, Constantin Van Outryve D'Ydewalle, Charlotte J. Sumner
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Abstract
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.
Claims21
A method of increasing SMN expression in a cell, comprising contacting the cell with an antisense compound, wherein the antisense compound comprises an oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the oligonucleotide is at least 85% complementary to a SMN-NAT nucleic acid sequence, wherein the SMN-NAT nucleic acid sequence is at least 85% identical to SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.