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New RNA-Snipping CRISPR Gene-Editing System May Help Treat Huntington's And Other Diseases

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With scientists learning more about the potential of CRISPR as a tool for gene-editing, it is now becoming increasingly possible to develop better forms of treatment that can target specific malignancies in the body.

One such recent breakthrough is the use of a CRISPR formed from mouth bacterium that is capable of breaking RNA, the part of cells that help transform genes into usable proteins.

If this technique proves successful, it could lead to better ways on how to modify the proteins in cells, or even eliminate cancer cells in humans.

The RNA version of CRISPR was developed by researchers at the Massachusetts Institute of Technology (MIT). It is based on a certain enzyme known as C2c2, which helps keep bacteria protected against other microbes such as viruses.

In a study featured in the journal Science, Dr. Feng Zhang and his colleagues at the Broad Institute at MIT described how they were able to engineer the C2c2 enzyme in order to search out and break apart specific RNA chemical sequences.

This is important as RNA serves as template to which the genetic codes from an individual's DNA is stored and transferred to the body's natural machinery for making proteins.

By manipulating the RNA, researchers could influence gene activity as well as the production of protein in the body. This would effectively grant them the ability to turn the process up or down, or even switch it on or off to suit their purposes without affecting the genetic codes stored in the RNA.

The new technique can provide scientists with a better understanding on the various biological pathways, which they can use to make better disease treatments.

"C2c2 opens the door to an entirely new frontier of powerful CRISPR tools," Dr. Zhang said.

"There are an immense number of possibilities for C2c2 and we are excited to develop it into a platform for life science research and medicine."

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