HUMBLE chemicals rather than advanced gene therapy strategies might be enough to treat some genetic diseases. That's the exciting implication of research in the US, which demonstrates that certain compounds can fix proteins that are produced by defective genes.
Some genetic diseases are caused by subtle defects in proteins that make them unstable at body temperature and unable to fold into the correct shape. Yet at lower temperatures, they will function normally. This led William Welch of the University of California in San Francisco to question the use of gene therapy to replace these proteins. "If it's only broken a little, why not try to fix it a little, rather than throw it out?" he says.
Welch knew that small molecules such as those of glycerol, "heavy" water containing deuterium, and trimethylamine N-oxide (TMAO) can stabilise proteins in a test tube or in bacteria, but no one had ...
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