New drug overrides 'nonsense' mutations

New drug overrides 'nonsense' mutations


CERTAIN forms of genetic disease could be beaten by overcoming so-called “nonsense” mutations that cause them. Nonsense mutations put “stop” signals in the middle of genes, causing cells’ protein-making machinery to finish early. The result is non-functional proteins that cause diseases such as cystic fibrosis and Duchenne muscular dystrophy (DMD). Lee Sweeney of the University of Pennsylvania in Philadelphia and colleagues previously found that the antibiotic gentamicin could overcome the mutations responsible for 15 per cent of DMD cases (New Scientist, 14 August 1999, p 21). It proved too toxic at the required levels,
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