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By Karin Kloosterman April 26, 2009
Antibiotics
are no doubt the wonder drug of the 20th century. Before them, people
could die from simple bacterial infections. Now, new groundbreaking
research from Israel shows that a common antibiotic has the power to
repair genetic diseases like cystic fibrosis, cancer and muscular
dystrophy.
The Israeli team headed by Prof. Timor Baasov of the Technion -
Israel Institute of Technology, has modified a common antibiotic, one
without toxic effects, and which is programmed to fix "nonsense"
mutations in genetic diseases. Nonsense mutations are mutations in a
sequence of DNA which causes it to prematurely stop reading the RNA,
resulting in an incomplete and non-functional protein being created.
Since releasing the news of the new compound, called NB54, based on the
common antibiotic gentamicin, Baasov has been flooded with queries from
investigators all over the globe excited to test it, he tells
ISRAEL21c. The findings were published online in March in the Journal of Medicinal Chemistry.
Gentamicin is from a group of antibiotics known as aminoglycosides,
which are used to treat a wide range of bacterial infections. It is
commonly used to treat enlarged prostate glands, chest infections,
urinary tract infections and infected wounds or burns. Previous studies
find gentamicin can work to counteract genetic diseases when mutations
cause disruptions of the development processes of proteins.
Compound working in cystic fibrosis
The drug enables ribosomes, which carry out protein synthesis, to
ignore these genetic disruptions and generate healthy, full-length
functional proteins instead.
But until now, doctors were not able to prevent the toxic side
effects needed for the treatment. Profound hearing loss was one of the
unwanted shortcomings of using this antibiotic for treatment.
"We have shown our lead compounds are more powerful than existing
antibiotics and much less toxic. We also have data on preclinical
studies on cystic fibrosis that it works well. This is the very new
direction that shows how chemistry can modify toxic compounds to be
useful as a drug," says Baasov.
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