Neutralizing Toxic RNA

摘要

Expansion of tandem nucle-otide repeats in the human genome-most often the triplets CAG and CTG-causes a number of inherited diseases such as Huntington's disease, spi-nocerebellar ataxia, and myotonic dystrophy. An expansion within the coding region of a gene can reduce the normal function of the corresponding protein and/ or cause an aberrant function (7), whereas expansions in noncoding regions can cause disease (such as myotonic dystrophy) without interfering with protein sequence. In myotonic dystrophy, RNA that is transcribed from DNA containing noncoding expansions is causative of disease pathogenesis. On page 336 of this issue, Wheeler et al. (2) present a potentially highly effective approach to neutralize the toxic effects of these RNAs.