ABSTRACT

CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease 9) provides powerful gene-editing tools that are applicable for gene therapy of a variety of diseases including, but not limited to cancer, rare diseases, and heart disease. In the current study, we first examined our artificial stem cell and organoid models that were generated by our literature validated DeepNEU platform from the perspective of gene-editing. We then evaluated the aiCRISPRL (aiCRISPR-Like) application of the DeepNEU platform by comparing the CRISPR-based gene-editing approach with the DeepNEU derived aiCRISPRL capabilities using artificial simulated HeLa cells (aiHeLa). We then evaluated the aiCRISPRL like capabilities of DeepNEU to introduce a series of specific mutations into the MutS homolog 2 (MSH2) gene to assess DNA Mismatch Repair (MMR). This approach permits a comparative assessment of CRISPR and aiCRISPRL technologies following the introduction of specific MSH2 mutations. When combined with our previous research the current data indicate that aiCRISPRL is an evolving AI platform technology that can be quickly and reliably deployed in gene therapy applications to complement wet-lab based gene-editing technologies.