The material is registered as a patent and may help cure hundreds of genetic diseases
Technion researchers have succeeded in developing a new substance for the treatment of genetic diseases, which may help cure hundreds of genetic diseases.
The new material was developed by Professor Timor Bazov from the Shulich Faculty of Chemistry at the Technion, in collaboration with Dr. Tamar Ben Yosef from the Rappaport Faculty of Medicine. They recently published the discovery in the scientific journal
Bioorganic and Medicinal Chemistry letters"".
"In every cell there are ribosomes, which are a kind of 'machines' that translate the genetic information into protein," explains Professor Bazov. "The substance we developed, NB30, binds to the human ribosome, and instead of having a stop mutation (a mutation that creates a break in protein translation), it puts another amino acid that allows translation to continue until an active protein is obtained."
Professor Bazov has been working for many years on a family of antibiotic drugs called aminoglycosides, which have a wide range of activity. Bacteria have begun to develop great resistance against them, and Professor Bazov is studying this resistance mechanism. "One of the problematic bacteria is a bacteria called Pseudomonas, which is much more resistant than Klebsiella," says Professor Bazov. "It especially attacks patients with a poor immune system. We developed antibiotic substances that were effective against this bacterium. In 2003 I met with Professor Eitan Kerem from the Hadassah Hospital in Jerusalem and it became clear to me that about 50% of cystic fibrosis patients get rid of infections caused by this bacterium. Professor Kerem proved that the antibiotic gentamicin can also be used to repair a damaged gene in cystic fibrosis patients, making it much easier for them. The problem is that it must be given in a high dose that could harm the patient, even to the point of death. At the Technion, we took gentamicin, chemically modified it, and created a new substance that we showed is also capable of repairing a damaged gene, and is less toxic (7 times) than gentamicin. The new material may be useful for curing hundreds of genetic diseases and possibly certain types of cancer as well."
