METHODS OF TREATING DISORDERS CAUSED BY FORMATION OF TRANSCRIPTS CARRYING NONSENSE MUTATIONS

摘要

A method of treating an individual having a disorder caused by formation of an RNA transcript carrying a nonsense mutation is provided. The method is effected by exposing cells of the individual expressing the RNA transcript to treatment selected capable of increasing a half life of the RNA transcript in the cells; and exposing the cells to an aminoglycoside thereby treating the individual having the genetic disease caused by formation of the RNA transcript carrying the nonsense mutation.