Repairing faulty genes

Israeli scientists have developed compounds that could be better treatments for genetic diseases than current drugs.

Timor Baasov and his colleagues at the Israel Institute of Technology have improved compounds used to suppress faults in genes called nonsense mutations.

Nonsense mutations, which cause more than 1800 human diseases, are alterations in the genetic code that stop protein production prematurely, leading to truncated or nonfunctional proteins. Gene therapy is one treatment, but it’s had limited success. With suppression therapy, small molecules allow cells’ protein producing equipment to skip over nonsense mutations to restore the proteins. Aminoglycosides – antibiotic amine-modified sugars – are the only clinically available drug family known to be effective in suppression therapy, but at effective doses, the compounds have high human toxicity.

Two aminoglycoside derivatives were found to suppress alterations in the genetic code called nonsense mutations

Read the full story in Chemistry World
Link to journal article
Repairing faulty genes by aminoglycosides: Identification of new pharmacophore with enhanced suppression of disease-causing nonsense mutations
Jeyakumar Kandasamy, Dana Atia-Glikin, Valery Belakhov and Timor Baasov,
Med. Chem. Commun., 2011, DOI: 10.1039/c0md00195c

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