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US 11,535,848 B2Granted
Compositions and methods for modulation of SMN2 splicing in a subject
AssigneeBiogen MA Inc.
InventorsFrank Rigo, C. Frank Bennett
Life of this patent
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Abstract
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject.
Claims12
An antisense oligonucleotide consisting of the nucleic acid sequence of SEQ ID NO:7, wherein all cytosine residues of the nucleic acid sequence are 5-methylcytosines, wherein each nucleoside of the nucleic acid sequence comprises a 2′-O-methoxyethyl sugar modification, and wherein the nucleic acid sequence has the following backbone chemistry:
- sossssossssossssoss, wherein “s” indicates a phosphorothioate linkage and “o” indicates a phosphodiester linkage.
Dependencies are inferred from the claim text.