AAV-Cas9 SmartNuclease™ System
rAAV Vectors for Efficient Cas9 Delivery In Vivo
- Deliver Cas9 in vivo
- Edit genomes in post-natal animals
- Develop gene therapies in small animal models
- Generate novel disease models
- Choose from all-in-one or two-vector AAV-Cas9 systems
AAV-Cas9 SmartNuclease System
Bringing together the versatile CRISPR/Cas9 genome editing system with powerful recombinant AAV (rAAV) technology, SBI’s AAV-Cas9 SmartNuclease vectors extend genome editing capabilities to cutting edge in vivo applications.
• Deliver Cas9 in vivo
• Edit genomes in post-natal animals
• Develop gene therapies in small animal models
• Generate novel disease models
• Choose from all-in-one or two-vector AAV-Cas9 systems
With their broad tropism, the lack of disease associated with wild-type virus, ability to transduce both dividing and non-dividing cells, and long term transgene expression, recombinant AAV (rAAV) has recently become the method of choice for delivering gene therapy and genome engineering vectors to intact organisms (Vasileva A, 2005; Petrs-Silva H, 2013). However, for efficient packaging, inserts into the region between rAAV’s two ITR sequences must be less than 5 kb.
The development of CRISPR/Cas9 has already revolutionized what’s possible when it comes to manipulating the genomes of even complex organisms. However, in vivo delivery via rAAV vectors has been hampered by the size of the Streptococcus pyogenes Cas9 gene (spCas9), the most widely-used form of Cas9. To overcome this problem, Ran, et al,1 characterized smaller orthologs of the Cas9 gene and found that Cas9 from Staphylococcus aureus (saCas9) performs as efficiently as spCas9 while being ~1 kb shorter, enabling insertion into rAAV vectors.
Why SBI for AAV-Cas9?
With advanced rAAV systems and a range of easy-to-use Cas9 vectors and kits, SBI has the expertise to combine these two technologies into a single, easy-to-use, and powerful system. Choose from our all-in-one or two-vector systems to drive your in vivo genome editing studies into high gear.
1. Ran, F. A. et al. In vivo genome editing using Staphylococcus aureus Cas9. Nature 520, 186–191 (2015).