CRISPR/spCas9 nuclease AAV AAV

Cas9 is the nuclease guided by the crRNA and tracrRNA (or trans-activating crRNA) to cleave specific DNA sequences. A guide RNA (gRNA) can be designed to include a hairpin that mimics the tracrRNA-crRNA complex. Binding specificity is based on the gRNA and a three nucleotide NGG sequence called the protospacer adjacent motif (PAM) sequence. With its ease in designing guide sequences to target specific genomic loci, the CRISPR/Cas system is a much simpler, faster, and robust alternative to TALEN and Zinc finger nuclease platforms.

The AAV-DJ is a synthetic serotype made from DNA family shuffling of 8 wild type serotypes of AAV, including AAV2, 4, 5, 8, 9, avian, bovine and goat AAV. This synthetic serotype has better infection efficiency in most cultured cells than wild type AAV serotype 1-8, and it also has superior liver & neuronal tropism for in vivo infection.

This is an AAV-DJ expressing spCas9 nuclease under a short CMV7 promoter. It can be used along with guided RNA for targeted genome-editing. We can also package this spCas9 into other serotypes of AAV upon request.