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CRISPR Based RNA Editors

  • HuidaGene’s CRISPR Cas13 X/Y

    Cas13 is a single RNA-guided RNA endonuclease to target RNA and has shown activities in targeted knockdown, RNA imaging, and RNA editing without mutating the genome. RNA editing has multiple advantages over more traditional DNA editing systems; first, RNA editing doesn't require homology-directed repair (HDR) machinery; thus, it could be used in non-dividing cells. Cas13 enzymes, unlike Cas9 and Cas12a (previously named Cpf1), also don’t require a protospacer adjacent motif (PAM) sequence at the target locus, making them more flexible than Cas9/Cas12a. Some Cas13 enzymes prefer targets with a given single base protospacer flanking site (PFS) sequence, but Cas13 enzymes lack the RuvC (recombination UV resolvase) and HNH (His-Asn-His) domains required for DNA cleavage, preventing them from directly editing the genome. A Cas13-based RNA editing system would likely be reversible, avoiding genomic off-targets or indels introduced by non-homologous end joining (NHEJ).

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Proprietary RNA Editing Tools

  • RNA Editor - hfCas13X/Y
    ⦁ RNA knockdown
    ⦁ Suitable for single AAV (<800aa)
    ⦁ High efficiency ( in vitro ~100%, in vivo >90%)
    ⦁ Low off-target
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  • RNA Base Editor – emxABE / emxCBE
    ⦁ Mutation correction (RNA base editing A-to-I & C-to-U)
    ⦁ Suitable for single AAV (<850aa);
    ⦁ High efficiency ( in vitro >90%, in vivo >90%)
    ⦁ Low off-target
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