Many genetic diseases, such as cystic fibrosis and muscular dystrophy, are caused by DNA mutations that terminate protein synthesis in the mutant gene. Aminoglycoside based antibiotics have been shown to stop these mutations but are often toxic and cause unpleasant side-effects in patients.
Now Timor Baasov and colleagues have designed and synthesised new aminoglycoside derivatives that show enhanced targeting and reduced cell toxicity, compared with the leading antibiotic gentamicin. Baasov’s compounds contain a chiral methyl group at the side-chain of the ribosamine ring, which they claim could be an essential feature responsible for the compound’s biological activity.
These findings could have immediate therapeutic applications for the treatment of many genetic diseases and could also aid treatment of several types of cancer caused by DNA mutations.
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Repairing faulty genes by aminoglycosides: Identification of new pharacophore with enhanced suppression of disease-causing nonsense mutations.
Jeyakumar Kandasamy, Dana Atia-Gilkin, Valery Belakhov and Timor Baasov
Med.Chem. Commun, 2011, Advance Article, DOI: 10.1039/C0md00195c