CRISPR delivery vehicle
Our proprietary CRISPR-Nanoparticle can directly deliver Cas9 RNP and donor DNA in vivo via local administration and induce HDR. CRISPR-Nanoparticle is composed of gold nanoparticles conjugated with DNA, which are complexed with donor DNA, Cas9 RNP, and the endosomal disruptive polymer. CRISPR-Nanoparticle has shown that they deliver Cas9 RNP and donor DNA into a wide variety of cell types and in vivo by direct injection.
Each component of CRISPR system can be engineered to enhance functionality or delivery. We have identified several gRNA and donor DNA chemical modifications that enhance cell delivery of CRISPR/Cas9 therapeutics, and also enable enrichment of gene edited cells. The chemical engineering of gRNA and donor has numerous applications to enhance the delivery efficiency and tissue specificity of CRISPR. Moreover, the approach can deliver donor DNA together with CRISPR just like one molecule so that HDR efficiency can be enhanced