COMPOUNDS AND METHODS FOR MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION

摘要

To provide methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal.SOLUTION: The present invention provides a composition that comprises a compound comprising a modified oligonucleotide consisting of 10-30 linked nucleosides and having a nucleobase sequence comprising a complementary portion containing at least 8 contiguous nucleobases complementary to a target region of equal length of a DMPK nucleic acid, a pharmaceutically acceptable carrier or diluent. The present invention also provides a method for reducing a DMPK mRNA in a cell, including bringing a cell into contact with the composition.SELECTED DRAWING: None