Genome editing can now be carried out in an isogenic setting, CRIS has great potential as a medical promise.
Moataz Dowaidar
Abstract
As a result of genome editing, the field of cardiovascular research and treatmentis changing. Related variants can now be introduced into patient-derived cellsand tested in an isogenic environment. To evaluate the safety and efficacy ofgene-editing therapeutics, cellular phenotyping of genome-edited iPSC-derivedcardiomyocytes and other cell types will be invaluable. Parallel to theseadvances, viral vectors and nanoparticles can be used to efficiently edit genes inthe liver and core. Somatic gene editing has been used to treathypercholesterolemia, hypertriglyceridemia, WPW syndrome,catecholaminergic polymorphic ventricular tachycardia, and Duchennemuscular dystrophy in animal models. While these early achievements arepromising, they have also shown major challenges. Off-target editing withCRISPR/Cas systems will be influenced by the same editing enzyme,architecture, target cell type, and delivery mechanism. It is possible to transmitefficiently to somatic tissues in mice, but not all delivery pathways scale well tohumans. On-target unintentional editing operations, such as major deletions andinsertions, require further analysis and risk evaluation. Immunity, as well as thesubsequent development of an immune response to the Cas9 protein extractedfrom bacteria, will necessitate caution. Despite these reservations, CRISPR/Cashas tremendous therapeutic promise, and it is expected to enhance research andmedical treatment in the future.