The CRISPR-Cas9 gene editing approach was found to protect against ischemia/reperfusion injury in a mouse study.
However, most gene editing strategies focus on correcting specific genetic mutations that occur only in a small proportion of patients and often before the onset of disease.
Gene therapy for cardiovascular disease
Broader applications of this approach remain limited. Here, Simon Lebeck, Eric Olson and colleagues present a CRISPR-Cas9 gene editing therapy that can be used to treat a range of heart disease patients. Ischemia/reperfusion (IR) injury is a type of tissue damage that occurs after a variety of cardiovascular injuries, including stroke and heart attack.
Chronic overactivation of Ca2+/calmodulin-dependent protein kinase IIδ (CaMKIIδ) is known to induce several cardiac diseases in humans and mice, including IR injury.
Oxidation of methionine residues causes CaMKIIδ hyperactivation. Lebek et al. found that using CRISPR-Cas9 adenine base editing to eliminate oxidative activation sites of the CaMKIIδ gene in cardiomyocytes protected them from IR injury in mouse models. In addition, Lebek et al. found that injecting gene-editing reagents into mice immediately after IR injury allowed the animals to recover cardiac function after severe injury.