High-precision gene therapy
Biologists have invented a selective switch of RNA therapy
Georgy Golovanov, Hi-tech+
Transgenes — DNA fragments introduced into the genome to change its properties — can have a negative or even harmful effect if they get into the wrong cells. Specialists from MIT and Harvard University has come up with a way to selectively launch gene therapy in patient cells. The technology makes it possible to detect specific sequences of matrix RNA, as a result of which the production of a specific protein from a transgene begins. This reduces the risk of errors in gene therapy.
mRNA molecules are strands of RNA that contain instructions for making certain proteins. A few years ago, Professor James Collins and his colleagues figured out how to use RNA detection as a trigger that stimulates the production of a specific protein in bacterial cells.
In a new study, scientists have tried to create a similar system that can be used in eukaryotic cells, including human cells. In order for the method to work for eukaryotic cells, they used the same system that viruses use to penetrate cells. It consists of regulatory sites of eukaryotic mRNA and their viruses, which are called IRES (internal ribosome entry sites, the site of the internal landing of the ribosome), tells MIT News.
"The basic idea is that you can select any unique RNA signature and deliver a drug," said Angelo Mao, lead author of the paper published in Nature Biotechnology. — This is a way to reduce the expression of a biomolecule to target cells or tissues."
The scientists took the natural IRES of various types of viruses and edited them by inserting chains that connect to the mRNA trigger. When such a site is introduced into a human cell before a transgen, it blocks the translation of this gene until a trigger mRNA is detected inside the cell.
Using this method, mRNA molecules have been created that can detect various triggers inside human and yeast cells. First of all, they were tested for Zika and SARS-CoV-2 viruses. Based on this technology, scientists propose to create T-cells capable of recognizing and responding to viral mRNAs during infection. In addition, the same method can be used to identify the condition of cells, as well as to detect cancer cells.
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