sacas9 d10a nickase

CRISPR/Cas9 nickase (SaCas9 D10A) is a genome-editing tool derived from the Staphylococcus aureus Cas9 protein, modified with a D10A mutation in the RuvC nuclease domain to convert it from a double-strand nuclease into a nickase. This enzyme creates a single-strand break (nick) in the target DNA rather than a double-strand break (DSB), which significantly reduces the frequency of non-homologous end joining (NHEJ)-mediated insertions and deletions (indels) and minimizes off-target effects. In therapeutic development for Duchenne muscular dystrophy (DMD), the SaCas9 D10A nickase is utilized in a single gRNA (SNSG) approach to precisely excise large gene duplications. By targeting the duplicated region, the system restores the wild-type DMD gene sequence and full-length dystrophin protein expression. The therapeutic is typically delivered using an adeno-associated virus (AAV) vector under the control of muscle-specific promoters like CK8e.

Other names

SaCas9 (D10A)Staphylococcus aureus Cas9 D10A nickaseCRISPR/Cas9 nickase (SaCas9 D10A)