Antisense RNAs against psoriasis
Psoriasis RNA therapy to be tested in humans
At the annual conference of the American Chemical Society, a report was made on the upcoming trial of a new method of treating psoriasis using specially created RNA molecules (Robert Service, Spherical RNA therapy shows promise against psoriasis in the first human trial).
Information about the synthesis of necessary proteins is recorded in human cells using DNA. It is read from the corresponding gene when a single-stranded information RNA molecule is synthesized. This molecule is sent from the cell nucleus to the ribosome, where the genetic code is converted into amino acids – the building material for proteins. In some diseases, too much of any necessary protein is produced, for example, with the overproduction of a protein called tumor necrosis factor - alpha (TNF–alpha), the immune system is overly stimulated and its activity leads to manifestations of psoriasis: redness and scaliness of the skin. To interrupt this process, so-called "antisense RNAs" can be used. These molecules bind on the principle of complementarity with informational RNAs and prevent their reading in ribosomes. Hence, the synthesis of the corresponding protein is reduced.
But getting RNA to work for medicinal purposes is difficult. Most often antisense RNAs are quickly destroyed by special enzymes that protect the cell from the penetration of alien genetic material. Therefore, today, despite decades of efforts and dozens of clinical trials, only two methods of RNA therapy are approved for use in the United States.
In the new method, they try to circumvent the difficulty by using not linear molecules, but so-called spherical nucleic acids (SNCs). Their molecule is densely packed identical fragments of nucleic acids on the spherical surface of the central object: metal nanoparticles or, as in the current case, a lipid. Such molecules more easily penetrate into cells through the membrane and are less susceptible to "persecution" by cellular enzymes.
The clinical trial of RNA therapy for psoriasis is conducted by Exicure, whose founder, Chad Mirkin, developed the first SNK molecules in 1996. The first phase of the test was able to confirm the safety of the molecules used for humans. Now it is necessary to check the effectiveness of this method and select the necessary dose of the drug. Robert Langer, a specialist in drug development from the Massachusetts Institute of Technology, believes that if the current trial is successful, not only Exicure, but also other manufacturers will develop methods of RNA therapy for many diseases.
Portal "Eternal youth" http://vechnayamolodost.ru
31.08.2016