Ishaan Jindal1, Xiao Wang2. 1. Cardiovascular Institute, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. 2. Cardiovascular Institute, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, 11-189 Smilow Center for Translational Research, 3400 Civic Center Boulevard, Bldg. 421, Philadelphia, PA, 19104, USA. xiao8@pennmedicine.upenn.edu.
Abstract
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.
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.
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