Advancing CRISPR Gene-Targeting Technology

Article

The CRISPR system may revolutionize the treatment of a range of diseases.

A characterization of a new CRISPR system is able to target RNA, allowing researchers to make temporary changes with greater specificity and functionality than current RNA methods.

C2c2 is an RNA-guided enzyme with the ability to target and degrade RNA, a study published in Science found. The system was found to help protect bacteria from viral infection, showing that C2c2 can be programed to splice specific RNA sequences in bacterial cells.

By specifically targeting RNA, it provides researchers with the ability to specifically manipulate RNA in a high-throughput manner, and more broadly manipulate gene function.

“C2c2 opens the door to an entirely new frontier of powerful CRISPR tools,” said senior study author Feng Zhang. “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.”

This process could help researchers gain further knowledge on different diseases and their treatment and prevention.

“The study of C2c2 uncovers a fundamentally novel biological mechanism that bacteria seem to use in their defense against viruses,” said senior study author Eugene Koonin. “Applications of this strategy could be quite striking.”

Current techniques for gene knockdown is small interfering RNA (siRNA); however, researchers believe that the C2c2 RNA editing method could be used for a wider range of applications including: adding molecules to specific RNA sequences to alter the function, making them valuable tools for large scale screens and creating synthetic regulatory networks; and harnessing C2c2 to tag RNAs fluorescently to help study their subcellular localization and trafficking.

Additionally, since C2c2 could remove specific RNA sequences, it could be a potential alternative approach to siRNA, allowing researchers to have adjustable gene knockdown abilities using RNA.

“C2c2's greatest impact may be made on our understanding the role of RNA in disease and cellular function,” said first co-author Omar Abudayyeh.

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