Compositions and methods for modulation of SMN2 splicing
AssigneeIsis Pharmaceuticals, Inc.
InventorsBrenda F. Baker, Adrian R. Krainer, Yimin Hua
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Abstract
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
Claims14
An antisense oligonucleotide targeted to intron 6 of a nucleic acid molecule encoding SMN2, wherein said oligonucleotide is 12 to 20 nucleotides in length and comprises a 2′-O-methoxyethyl sugar modification at each position, and wherein said oligonucleotide is targeted to an intronic splicing silencer element.
Dependencies are inferred from the claim text.