Abstract
The novel gene editing technology CRISPR‐Cas9 shows great promise in revolutionizing treatments of genetic disorders; however, currently the only practical treatments are ex vivo, limited to blood diseases. In vivo treatment can substantially extend the clinical reach of the technology, but is limited by a challenging delivery process. The large size of the Cas9 gene (~4500bp) inhibits delivery by the small viral vectors and lipid nanoparticles used. We hypothesize that a Cas9 gene codon optimized for human cells will support significantly higher protein expression than the presently used genes; this can compensate for the low delivery efficiency and has great potential to improve the viability of in vivo CRISPR‐Cas9 therapies.To investigate our hypothesis, we selected a commonly used parent saCas9 Addgene’s public plasmid database. We engineered a novel, codon‐optimized gene sequence from the parent using GenScript’s OptimumGene™ algorithm. We will compare protein expression via ELISA assay and Western Blot analysis. In this poster we will present the data gleaned from these assays.
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