According to the latest reports, it seems that engineers can selectively turn on gene therapies in human cells. Here are more details about the mind-blowing issue.
Turning on gene therapy
A team of researchers from MIT and Harvard University has developed a method to activate gene therapies selectively in target cells, including human cells.
The technology can identify specific messenger RNA sequences present in the cells, which then triggers the production of a specific protein from an artificial gene or transgene.
Gene therapies can have negative or even harmful effects when expressed in the wrong cells. To minimize off-target effects from gene therapies, the researchers sought to differentiate various types of cells by examining the RNA sequences present in them. These RNA sequences differ from tissue to tissue.
The researchers have developed a new technology that could improve gene therapies for various applications, such as regenerative medicine and cancer treatment.
This technology allows the production of transgene only after “reading” specific RNA sequences within cells. With this fine-tuning capability, researchers could potentially design new therapies to target cancer cells by identifying and producing toxic proteins only inside those cells, leading to their destruction.
“This brings new control circuitry to the emerging field of RNA therapeutics, opening up the next generation of RNA therapeutics that could be designed to only turn on in a cell-specific or tissue-specific way,” says James Collins, the Termeer Professor of Medical Engineering and Science in MIT’s Institute for Medical Engineering and Science (IMES) and Department of Biological Engineering and the senior author of the study.
The researchers have developed a new approach that focuses on a genetic element used by viruses to regulate gene translation in host cells.
This method is highly targeted and may help to avoid some of the side effects associated with therapies that affect the entire body.
The study was led by Evan Zhao, a research fellow at the Wyss Institute for Biologically Inspired Engineering at Harvard University, and Angelo Mao, an MIT postdoc and technology fellow at the Wyss Institute, and was published on October 28, 2021, in Nature Biotechnology.
