Purpose of review: To establish genome editing as a promising therapeutic approach for the treatment and prevention of atherosclerotic cardiovascular disease.
Recent findings: Systemic delivery of a CRISPR adenine base editor using lipid nanoparticles demonstrated a near 90% reduction in circulating PCSK9 and over 60% reduction in blood LDL-C in nonhuman primates with the effects remaining durable at least 8 months following a single course. Preclinical proof-of-concept studies have elucidated the superior therapeutic potential of genome-editing approaches for the treatment of hyperlipidemia, thus substantiating their progression to clinical studies.
Keywords: Atherosclerotic cardiovascular disease; CRISPR/Cas9; Genome editing; High cholesterol; Hyperlipidemia; One-and-done therapy.
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