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New Gene Editing Method Discovered

Researchers discover a new method to selectively delete genes products that are responsible for causing certain diseases, according to a new study published on May 29, 2018.

A team of researchers at the Scripps Research Institute campus in Florida developed a new approach of treatment or cure of diseases by editing the genes driving these disease. Technologies such as CRISPR-Cas9, a protein-based system is one such gene editing techniques, which researchers have successfully managed to find an alternative for.

The new technique is a small-molecule-based tool that affects the RNA in order to selectively delete certain gene products. This approach creates the potential for scientists to develop drugs that correct genetic diseases for patients suffering from them. These drugs would function in a way that allows them to destroy the toxic genes as well as control the defense mechanisms of the body.

Lead author of the study, Matthew D. Disney, PhD said, “These studies, like much science, were about a decade in the making. We are very excited to see how this initial application evolves. This research further shows that RNA is indeed a viable target to make medicines.”

The team explained that around 2 percent of our genome encodes proteins, while 70 to 80 percent of the genome is transcribed into RNA. This in turn, widens the scope for potential druggable targets.

“Anchoring our previous work with Targaprimir-96 to the targeted recruitment of RNase L, we were able to program the RIBOTACs approach to only degrade cells that highly express the miRNA-96 oncogene, thus allowing FOXO1 to signal the selective destruction of triple negative breast cancer cells,” said Costales, co-author of the study.

The researchers took advantage of RNA degradation system of cells to evoke the body’s ability to kill its own cancer. The RIBOTAC technology has potentially broad applications for cancer and other gene-driven diseases as well, he says.

Disney’s scientists also developed InfornaTM, which is a computational method to match RNAs adequate stability and structure to small, drug-like molecules that have the ability to bind the two. Targaprimir-96 and several other disease-modifying compounds, were developed by this technique.