Silencing unhealthy alleles naturally

摘要

Recently, it has been reported that small interfering RNAs can silence mutant alleles of genes while not affecting wild-type alleles. This high specificity should also limit off-target effects when these molecules are used in drug-target validation or as therapeutics. Small RNA technology is both extremely versatile, because virtually any expressed gene can be inhibited, and effective, because it exploits natural cellular machinery that evolved to use small RNAs to regulate gene expression.