Suppression of endogenous gene and replacement of suppressed gene sequence

摘要

The use of a suppressing endogenous gene product in the manufacture of a medicament for treatment of autosomal dominant diseases by introducing a replacement gene. The gene is introduced by: 1) providing suppressing nucleic acids or other suppression effectors able to bind to an endogenous gene, gene transcript or gene product to be suppressed, and 2) providing genomic DNA or cDNA (complete or partial) encoding a replacement gene wherein the suppressing nucleic acids are unable to bind to equivalent regions in the genomic DNA or cDNA to prevent expression of the replacement gene. The replacement nucleic acids have modifications in one or more third base (wobble) positions such that replacement nucleic acids still code for the wild type or equivalent amino acids.