Antisense modulation of superoxide dismutase 1, soluble expression
View Patent ↗Antisense compounds, compositions and methods are provided for modulating the expression of superoxide dismutase 1, soluble. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding superoxide dismutase 1, soluble. Methods of using these compounds for modulation of superoxide dismutase 1, soluble expression and for treatment of diseases associated with expression of superoxide dismutase 1, soluble are provided.
1. A compound 8 to 50 nucleobases in length targeted to the coding region of a nucleic acid molecule encoding human superoxide dismutase 1, soluble (SEQ ID NO:3), wherein said compound comprises at least an 8-nucleobase portion of SEQ ID NO. 15.
2. The compound of claim 1 which is an antisense oligonucleotide.
3. The compound of claim 2 wherein the antisense oligonucleotide comprises at least one modified internucleoside linkage.
4. The compound of claim 3 wherein the modified internucleoside linkage is a phosphorothioate linkage.
5. The compound of claim 2 wherein the antisense oligonucleotide comprises at least one modified sugar moiety.
6. The compound of claim 5 wherein the modified sugar moiety is a 2′-O-methoxyethyl sugar moiety.
7. The compound of claim 2 wherein the antisense oligonucleotide comprises at least one modified nucleobase.
8. The compound of claim 7 wherein the modified nucleobase is a 5-methylcytosine.
9. The compound of claim 2 wherein the antisense oligonucleotide is a chiracric oligonucleotide.
10. A composition comprising the compound of claim 1 and a pharmaceutically acceptable carrier or diluent.
11. The composition of claim 10 further comprising a colloidal dispersion system.
12. The composition of claim 10 wherein the compound is an antisense oligonucleotide.
13. An antisense compound 8 to 50 nucleobases in length targeted to the coding region of a nucleic acid molecule encoding human superoxide dismutase 1, soluble (SEQ ID NO;3), wherein said compound comprises at least an 8-nucleobase portion of SEQ ID NO. 13 or SEQ ID NO. 15 and wherein the antisense oligonucleotide comprises at least one modified sugar moiety.
14. The compound of claim 13 wherein the antisense oligonucleotide comprises at least one modified internucleoside linkage.
15. The compound of claim 14 wherein the modified internucleoside linkage is a phosphorothioate linkage.
16. The compound of claim 14 wherein the modified sugar moiety is a 2′-O-methoxyethyl sugar moiety.
17. The compound of claim 14 wherein the antisense oligonucleotide comprises at least one modified nucleobase.
18. The compound of claim 17 wherein the modified nucleobase is a 5-methylcytosine.
19. The compound of claim 14 wherein the antisense oligonucleotide is a chimeric oligonucleotide.
20. A composition comprising the compound of claim 14 and a pharmaceutically acceptable carrier or diluent.
21. The composition of claim 20 further comprising a colloidal dispersion system.
22. The composition of claim 20 wherein the compound is an antisense oligonucleotide.