RNA leads the way!

The CRISPR/Cas9 is a system that is used to inactivate or correct certain genes. The system has a downfall, however, because once it is turned on, it stays on and keeps editing genes. This is no bueno because it can cause editing of genes that are not supposed to be cut or corrected.

The original CRISPR/Cas9 system uses a guide RNA that matches the desired sequence and directs the Cas9 enzyme to the location on the gene where it will perform gene editing.

Researchers from at the University of California have developed a new method using “chemically modified RNA base-drugs” that are still able to direct the Cas9 enzyme to the gene for editing. What’s different is that this new RNA is able to activate and inactivate the Cas9 enzyme as needed (or wanted) by the researcher.

The modified RNA stops the Cas9 enzyme’s function when it runs out. Without the presence of the modified RNA, the Cas9 is unable to continue editing genes. Which is what they want! They are also trying a different method where a second modified guide RNA is incorporated and its main role is to shut off the first modified guide RNA. By shutting off the first guide RNA, the CRISPR/Cas9 system is also turned off. This is beneficial for understanding and potentially finding a solution to certain nervous system diseases that are affected by this mechanism.

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