hfCas12Max guide RNA (hfCas12Max gRNA)

The hfCas12Max guide RNA is the programmable RNA component of the high-fidelity Cas12Max (hfCas12Max) CRISPR system, a proprietary genome-editing platform developed by Arbor Biotechnologies (Arbor Biotechnologies, 2022). This guide RNA, specifically a CRISPR RNA (crRNA), is responsible for directing the hfCas12Max nuclease to a specific 20-24 nucleotide DNA sequence adjacent to a 5'-TTN Protospacer Adjacent Motif (PAM) (Al-Shayeb et al., 2022). It functions by forming a ribonucleoprotein (RNP) complex with the hfCas12Max protein, which then scans the genome for a matching target sequence. The hf designation indicates a high-fidelity variant engineered to minimize off-target editing, which is a critical requirement for clinical safety in human therapeutics (Arbor Biotechnologies, 2023). Once the gRNA base-pairs with the target DNA, the associated nuclease induces a double-strand break, triggering cellular DNA repair pathways for gene knockout or correction. This system is highly efficient in mammalian cells and is being developed for therapeutic applications in genetic disorders, such as liver-directed therapies and oncology (DeWeerd et al., 2023). Its compact size compared to traditional Cas9 proteins allows for more efficient delivery using viral vectors like adeno-associated virus (AAV). The specificity of the gRNA-DNA interaction is the primary determinant of the therapy's precision, making the design of the guide RNA central to the drug's efficacy and safety profile.

Other names

hfCas12Max crRNAhigh-fidelity Cas12Max guide RNACRISPR RNA for hfCas12Max

The guide RNA provides sequence-specific targeting for the hfCas12Max nuclease, enabling precise DNA cleavage at a complementary genomic locus.