Cas13

Cas13 is a CRISPR-associated enzyme that targets RNA. Unlike the DNA-targeting Cas9, Cas13 utilizes a single RNA-guided endonuclease to bind and cleave specific RNA sequences. It employs two distinct ribonuclease activities: one for processing its own CRISPR RNA (crRNA) and another for degrading the target RNA.

The system's specificity allows for the correction of mutations at the transcript level. For example, it has been used to repair KRAS-G12D mRNA in pancreatic cancer models with high efficiency while minimizing effects on healthy cells. It has been adapted into tools such as the REPAIR platform, which edits RNA bases to treat genetic disorders, including Usher syndrome, in animal models. Cas13 also possesses collateral RNA-cleavage activity, which is utilized in diagnostic platforms like SHERLOCK to detect pathogens, tumor DNA, and viral variants with high sensitivity. Its PAM-independent targeting and reduced off-target effects make it suitable for RNA imaging, phage genome engineering, and transient gene regulation.

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