Modified antibiotics could treat genetic diseases
7 May 2009
By modifying the properties of the common antibiotic gentamicin,
researchers at the Technion-Israel Institute of Technology have
developed what could become an effective treatment for many human
genetic diseases, including cystic fibrosis (CF), Duchenne muscular
dystrophy, Usher Syndrome and numerous cancers. The findings have been
published in the Journal of Medicinal Chemistry.
Gentamicin belongs to a class of antibiotics called aminoglycosides,
which are used to treat a wide range of bacterial infections. Studies
have shown that gentamicin can counteract genetic diseases, including
those mentioned above that occur when mutations cause disruptions of the
development processes of proteins.
The drug enables ribosomes (the structures within a cell that carry
out protein synthesis) to ignore these disruptions and instead generate
healthy, full-length functional proteins.
But using gentamicin to treat these diseases requires much higher
doses than those commonly prescribed for bacterial infections. At these
higher doses gentamicin is non-selective and extremely toxic to humans,
with irreversible hearing loss (ototoxicity) being the main negative
In search of a way to bypass these complications, the team led by
Professor Timor Baasov of the Technion Faculty of Chemistry modified
existing aminoglycoside antibiotic drugs, and carefully monitored
biological and toxicity tests of the resulting derivatives. The result
is “NB54,” a new (and patented) chemical derivative of gentamicin.
“We’ve created a new purpose for aminoglycosides by removing their
traditional, natural actions as antibiotics,” said Baasov. “The loss of
their antibacterial activity makes them highly selective, less toxic,
and allows for their use in repairing ‘wrong’ genes in human beings.”
So far, the researchers have observed the action of NB54 in ex vivo
cell lines. They are currently awaiting data from animal model testing.
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