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CRISPR-Cas9 gene editing is emerging as a prospective therapy for genomic mutations. However, current editing approaches are directed primarily toward relatively small cohorts of patients wit
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Ablation of CaMKIIδ oxidation by CRISPR-Cas9 base editing as a therapy for cardiac disease - Science
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A novel CRISPR-Cas9 approach that targets a damaging signaling pathway in the heart confers protection against ischemia and reperfusion injury, according to a study in mice. The findings suggest that gene editing could offer a permanent, advanced strategy for treating heart disease and even serve as an intervention to repair heart damage immediately after a heart attack. CRISPR-Cas9 gene editing has shown promise as a therapeutic approach for the treatment of rare inherited diseases. Chronic overactivation of Ca2+/calmodulin-dependent protein kinase IIδ (CaMKIIδ) is known to cause several heart diseases in humans and mice, including lesions. The researchers 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, the researchers found that injecting gene-editing reagents into mice shortly after IR injury allowed the animals to recover cardiac function after severe damage.