Biomaterials for gene editing therapeutics

Abstract The unique advantage of gene therapy is its specific and precise regulation of the genes that directly cause a disease. Due to its distinctive features, gene editing has emerged as a powerful tool for genetic engineering. A programmable nuclease can edit the genomic region of interest, facilitating the permanent inactivation of gene expression. Many early studies focused on the ability of gene editing technology to treat intractable genetic disorders. More recent studies, however, have evaluated the ability of gene editing technology to treat cancer with gene editing targeting oncogenes and immunosuppressive genes. As with other gene therapies, gene editing requires a strategy for delivery to the desired tissue and cell. Although viral vectors have shown excellent clinical efficiency and feasibility, nonviral vectors based on biomaterials have been actively studied because of their safety, diversity, and possibility of selection. This chapter introduces the principle of gene editing technology and delivery and examines the current state of research on gene editing using biomaterials.