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 in

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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