Ablation of CaMKIIδ oxidation by CRISPR-Cas9 base editing as a therapy for cardiac disease

S Lebek, F Chemello, XM Caravia, W Tan, H Li, K Chen… - Science, 2023 - science.org
S Lebek, F Chemello, XM Caravia, W Tan, H Li, K Chen, L Xu, N Liu, R Bassel-Duby…
Science, 2023science.org
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 with specific mutations. Here, we describe a cardioprotective strategy potentially
applicable to a broad range of patients with heart disease. We used base editing to ablate
the oxidative activation sites of CaMKIIδ, a primary driver of cardiac disease. We show in
cardiomyocytes derived from human induced pluripotent stem cells that editing the CaMKIIδ …
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 with specific mutations. Here, we describe a cardioprotective strategy potentially applicable to a broad range of patients with heart disease. We used base editing to ablate the oxidative activation sites of CaMKIIδ, a primary driver of cardiac disease. We show in cardiomyocytes derived from human induced pluripotent stem cells that editing the CaMKIIδ gene to eliminate oxidation-sensitive methionine residues confers protection from ischemia/reperfusion (IR) injury. Moreover, CaMKIIδ editing in mice at the time of IR enables the heart to recover function from otherwise severe damage. CaMKIIδ gene editing may thus represent a permanent and advanced strategy for heart disease therapy.
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