Many of the most difficult diseases to treat have a genetic basis: genetic diseases, of course, but also tumors and viral infections. Scientists have long been working on drugs that repair faulty genes, yet few of them are available on the market. Why? Researchers at Northeastern University have come up with an answer.
Professor Ke Zhang's team has developed a way to increase the efficacy of a class of these drugs, oligonucleotides. It is in fact frequent that the liver and kidneys eliminate the drugs of this class, rendering them ineffective. Researchers are therefore elaborating a defensive structure for the molecule, which isolates it and allows it to act on the organism. How does this affect the future of genetic medicine?
The shield consists of multiple chains of polyethylene glycol attached to the oligonucleotides. Chains make the drug too large to be filtered, protect it from enzymes and allow it to interact with genes. In this way the drug stays in circulation longer and has more time to destroy damaged genes. This could give new hope to many treatments, wrecked because the drug could not remain in circulation.
The team tested the shield on an anticancer medicine. Most of the oligonucleotides have attacked the genetic material of cancer cells. As a result, the rate of tumor growth has decreased and there has been an improvement in the health of the guinea pigs.