RNA editing using CRISPRs shows promise for genetic disease treatment

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RNA editing using CRISPRs shows promise for genetic disease treatment
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A team at Montana State University published research this week that shows how RNA, the close chemical cousin to DNA, can be edited using CRISPRs.

Apr 29 2024Montana State University The work reveals a new process in human cells that has potential for treating a wide variety of genetic diseases.

In our previous work, we used type-III CRISPRs to edit viral RNA in a test tube. But we wondered, can we program manipulation of RNA in a living human cell?" "We were confident that we could use these CRISPR systems to cut RNA in a programmable manner, but we were all surprised when we sequenced the RNA and realized that the cell had stitched the RNA back together in a way that removed the mutation," said Wiedenheft."The general belief is that there's not much point in repairing RNA," he said. "We speculated that RNA would be repaired in living human cells, and it turned out to be true.

RNA editing has important applications in the search for treatments of genetic diseases, Nemudryi said. RNA is a temporary copy of a cell's DNA, which serves as a template. Manipulating the template by editing DNA could cause unwanted and potentially irreversible collateral changes, but because RNA is a temporary copy, he said, edits made are essentially reversible and carry far less risk.

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