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This New RNA Editing Technique Could Help Delete Some Diseases

Scientists have been using the CRISPR gene-editing tool to alter DNA, but now a team of scientists from the Broad Institute claims to have expanded this tool to also edit RNA.

RNA is different to DNA in a variety of ways. While DNA is responsible for storing and transferring genetic information, RNA within the cells is used to transmit information coded within DNA to produce proteins. It features ribose sugars rather than deoxyribose sugars. It acts as an essential middle-man for any cell to use the instructions in the DNA. RNA degrades after about 24 hours, and according to scientists, editing RNA— theoretically—could help heal short-term conditions like inflammation.

The system developed by Broad Insitute is called “RNA Editing for Programmable A to I Replacement” or REPAIR.

The REPAIR system relies on enzyme Cas13 (instead of Cas9 normally used by CRISPR).

“REPAIR presents a promising RNA editing platform with broad applicability for research, therapeutics and biotechnology,” the researchers wrote in the study published in the journal Science.

So far, scientists have tested REPAIR in mammalian cells, and they hope to test the new system with humans too.

“The ability to correct disease-causing mutations is one of the primary goals of genome editing,” senior author Feng Zhang said.

“So far, we’ve gotten very good at inactivating genes, but actually recovering lost protein function is much more challenging. This new ability to edit RNA opens up more potential opportunities to recover that function and treat many diseases, in almost any kind of cell,” said Zhang.

In another breakthrough study carried out by a separate team from the Broad Institute and published in journal Nature, researchers announced to have got success in editing single points of DNA. Researchers revealed that they can now change the nitrogen bases that make up DNA, known as adenine (A), thymine (T), guanine (G) and cytosine (C).

Broad Institute is a biomedical study facility being run as collaboration between the Massachusetts Institute of Technology and Harvard University.