Syn-SaCas9HF AAV (Serotype 9)

Syn-SaCas9HF AAV (serotype 9) is a specially designed vector for delivery and expression of high-fidelity Staphylococcus aureus Cas9 (SaCas9) controlled by the human synapsin promoter. The AAV9 serotype is known for its ability to efficiently transfect a variety of tissues, including the central nervous system (CNS), liver, and muscle tissue, making it a valuable vector for delivering genetic material. AAV9 is particularly advantageous when targeting the central nervous system (CNS) because it can penetrate the blood-brain barrier, which is key to treating neurological diseases.

SaCas9 is a smaller alternative to the more common Streptococcus pyogenes SpCas9. The small size of SaCas9 makes it ideal for packing into the limited capacity of AAV capsids. In addition, the high-fidelity (HF) variant of SaCas9 has been carefully designed to improve editing precision by minimizing off-target effects, thus solving a significant problem in CRISPR-mediated genome editing. Expression from the human synapsin promoter ensures that SaCas9 is primarily active in neuronal cells. This neuronal-specific activity is ideal for research and therapeutic strategies aimed at correcting genetic abnormalities in neural tissue. The synapsin promoter enables selective and stable expression in neurons, ensuring that gene editing tools work where they are most needed without generating unintended activity in non-neuronal cells.