Compounds and methods for modulating smn2
AssigneeIonis Pharmaceuticals, Inc.
InventorsFrank Rigo, Thazha P. Prakash, Kar Yun Karen Ling, W. Brad Wan, William John Drury, III
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Abstract
Provided are compounds, methods, and pharmaceutical compositions for modulating SMN2 RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disorder. Such symptoms include reduced muscle strength; inability or reduced ability to sit upright, to stand, and/or walk; reduced neuromuscular activity; reduced electrical activity in one or more muscles; reduced respiration; inability or reduced ability to eat, drink, and/or breathe without assistance; loss of weight or reduced weight gain; and/or decreased survival.
Claims31
A modified oligonucleotide according to the following chemical structure:
- or a salt thereof.
A modified oligonucleotide according to the following chemical structure:
An oligomeric compound comprising a modified oligonucleotide according to the following chemical notation:
- mCns Ano mCns Tno Tns Tns mCns Ans Tns Ans Ans Tns Gns mCns Tns Gns Gns mCn (SEQ ID NO:
- 21), wherein:
- A=an adenine nucleobase,
- mC=a 5-methyl cytosine nucleobase,
- G=a guanine nucleobase,
- T=a thymine nucleobase,
- n=a 2′-NMA sugar moiety,
- s=a phosphorothioate internucleoside linkage, and
- o=a phosphodiester internucleoside linkage.
Dependencies are inferred from the claim text.