TBG-SaCas9 AAV (Serotype 9)

TBG-SaCas9 AAV Serotype 9 is a cutting-edge tool designed for genome editing applications that leverages the power of the CRISPR-Cas9 system. SaCas9, or Staphylococcus aureus Cas9, is a CRISPR-associated protein derived from Staphylococcus aureus. Similar to SpCas9 from Streptococcus pyogenes, SaCas9 is used for targeted genome editing. It recognizes and induces double-strand breaks at specific DNA sequences by forming a complex with a guide RNA (gRNA), resulting in precise genome modification. SaCas9 has the significant advantage of being smaller than SpCas9, which is very beneficial in in vivo applications where Cas9 protein and gRNA need to be delivered to cells or organisms. The smaller size of SaCas9 makes it easier to package into delivery vectors such as AAV, which have limited capacity for genetic material.

The use of the TBG (thyroid hormone binding globulin) promoter in this AAV system enables liver-specific expression of SaCas9, making this construct particularly suitable for liver-targeted gene therapy applications. Serotype 9 AAV vectors are known for their ability to transduce a variety of tissues, including the nervous system and heart tissue, but the TBG promoter significantly enhances its specificity for liver tissue. By delivering the SaCas9 enzyme together with specific guide RNA, the system can introduce double-strand breaks at the target genomic site, thereby achieving precise gene editing, gene knockout, or correction of various disease-related gene mutations.