PCV-Cas9

PCV-Cas9 is an engineered CRISPR/Cas9 system designed to enhance the precision of genome editing by promoting Homology Directed Repair (HDR). It consists of a Porcine Circovirus 2 (PCV) Rep protein DNA-binding domain fused to either wild-type Cas9 (PCV-wtCas9) or a nickase variant (PCV-nCas9). This fusion protein allows for the covalent tethering of a single-stranded oligodeoxynucleotide (ssODN) donor template directly to the Cas9-guide RNA ribonucleoprotein (RNP) complex. By spatially and temporally co-localizing the double-strand break (DSB) machinery with the repair template, the system shifts the DNA repair balance away from Non-Homologous End Joining (NHEJ) toward precise HDR. It is being investigated by researchers at the Institute of Bioorganic Chemistry, Polish Academy of Sciences, primarily for the correction of the point mutation in the β-globin gene associated with Sickle Cell Disease (SCD).

Other names

Porcine Circovirus 2 Rep protein-Cas9 fusion