Gene editing primarily focus on threshold of edited protein as an Index of success rate. However in almost all cases, gene editing is met with severe liver edema and (or) other cellular toxicities. In most cases the immunogenicity is contributed to Cas9 protein, however the role of RNA secondary structure in the spikes of immunogenicity is often underestimated.
Simple zebrafish screens can point out at picogram level sensitivity, if RNA sequence based secondary structures are safe or not.
Additionally Pentagrit's screening system uses the relevant zebrafish disease model for the screening. This replicates similar immune and proteome profile as the patient who will be receiving the gene therapy.
Gene Editing can be safer and efficient with Pre-Clinical Zebrafish Assays.
Reach out to Viji@pentagrit.com for research queries.