Genome Editing Technology: A New Frontier for the Treatment and Prevention of Cardiovascular Diseases

Abstract

Over the past 2 decades, genome-editing technique has proven to be a robust editing method that revolutionizes the field of biomedicine. At the genetic level, it can be efficiently utilized to generate various disease-resistance models to elucidate the mechanism of human diseases. It also develops an outstanding tool and enables the generation of genetically modified organisms for the treatment and prevention of various diseases. The versatile and novel clustered regularly interspaced short palindromic repeats (CRISPR/Cas9) system mitigates the challenges of various genome editing techniques such as zinc-finger nucleases, and transcription activator-like effector nucleases. For this reason, it has become a ground-breaking technology potentially employed to manipulate the desired gene of interest. Interestingly, this system has been broadly utilized due to its tremendous applications for treating and preventing tumors and various rare disorders; however, its applications for treating cardiovascular diseases (CVDs) remain in infancy. More recently, 2 newly developed genome editing techniques, such as base editing and prime editing, have further broadened the accuracy range to treat CVDs under consideration. Furthermore, recently emerged CRISPR tools have been potentially applied in vivo and in vitro to treat CVDs. To the best of our knowledge, we strongly enlightened the applications of the CRISPR/Cas9 system that opened a new window in the field of cardiovascular research and, in detail, discussed the challenges and limitations of CVDs. 2023 Elsevier Inc.