Antisense modulation of superoxide dismutase 1, soluble expression

Abstract

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 . An antisense compound 8 to 50 nucleobases in length targeted to nucleobases 96-523 of a coding region of a nucleic acid molecule encoding human superoxide dismutase 1, soluble (SEQ ID NO: 3), wherein said compound specifically hybridizes with and inhibits the expression of human superoxide dismutase 1, soluble (SEQ ID NO: 3).  
     
     
         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 chimeric oligonucleotide.  
     
     
         10 . A method of inhibiting the expression of superoxide dismutase 1, soluble in brain and spinal cord, comprising intraventricularly administering to an animal a compound 8 to 50 nucleobases in length targeted to a nucleic acid molecule encoding superoxide dismutase 1 so that expression of superoxide dismutase 1, soluble is inhibited.