Compositions for modulating SOD-1 expression
Disclosed herein are antisense compounds and methods for decreasing SOD-1 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate SOD-1 associated diseases, disorders, and conditions. Such SOD-1 associated diseases include amyotrophic sclerosis (ALS).
1. A salt of an antisense compound, wherein the anion of the antisense compound has the following formula:
2. An antisense compound according to the following formula: mCes Aeo Ges Geo Aes Tds Ads mCds Ads Tds Tds Tds mCds Tds Ads mCeo Aes Geo mCes Te (nucleobase sequence of SEQ ID NO: 725); wherein
A=an adenine,
mC=a 5-methylcytosine,
G=a guanine,
T=a thymine,
e=a 2′-O-methoxyethylribose modified sugar,
d=a 2′-deoxyribose sugar,
s=a phosphorothioate internucleoside linkage, and
o=a phosphodiester internucleoside linkage,
or a salt thereof.
3. A composition comprising the antisense compound or salt thereof of claim 2 and at least one of a pharmaceutically acceptable carrier or diluent.
4. A composition comprising the salt of the antisense compound of claim 1 and at least one of a pharmaceutically acceptable carrier or diluent.
5. The composition of claim 3 , wherein the pharmaceutically acceptable carrier or diluent is phosphate-buffered saline (PBS).
6. The composition of claim 4 , wherein the pharmaceutically acceptable carrier or diluent is PBS.
7. The composition of claim 3 , which is formulated for intrathecal administration.
8. The composition of claim 4 , which is formulated for intrathecal administation.
9. The antisense compound of claim 2 .
10. The salt of the antisense compound of claim 2 .
11. A composition comprising the antisense compound of claim 9 and at least one of a pharmaceutically acceptable carrier or diluent.
12. The composition of claim 11 , wherein the pharmaceutically acceptable carrier or diluent is PBS.
13. The composition of claim 11 , which is formulated for intrathecal administration.
14. A composition comprising the salt of the antisense compound of claim 10 and at least one of a pharmaceutically acceptable carrier or diluent.
15. The composition of claim 14 , wherein the pharmaceutically acceptable carrier or diluent is PBS.
16. The composition of claim 14 , which is formulated for intrathecal administration.
17. An antisense compound according to the following formula:
or a salt thereof.
18. A composition comprising the antisense compound or salt thereof of claim 17 and at least one of a pharmaceutically acceptable carrier or diluent.
19. The composition of claim 18 , wherein the pharmaceutically acceptable carrier or diluent is PBS.
20. The composition of claim 18 , which is formulated for intrathecal administration.
21. The antisense compound of claim 17 .
22. A composition comprising the antisense compound of claim 21 and at least one of a pharmaceutically acceptable carrier or diluent.
23. The composition of claim 22 , wherein the pharmaceutically acceptable carrier or diluent is PBS.
24. The composition of claim 22 , which is formulated for intrathecal administration.