可编程基因组编辑技术作为动脉粥样硬化性心血管疾病的单疗程治疗方法。
Programmable Genome-Editing Technologies as Single-Course Therapeutics for Atherosclerotic Cardiovascular Disease.
机构信息
Cardiovascular Institute, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
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.
出版信息
Curr Atheroscler Rep. 2022 Nov;24(11):861-866. doi: 10.1007/s11883-022-01063-1. Epub 2022 Aug 22.
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.
目的综述
将基因组编辑确立为治疗和预防动脉粥样硬化性心血管疾病的一种很有前途的治疗方法。
最新发现
使用脂质纳米粒系统递送 CRISPR 腺嘌呤碱基编辑器，在非人类灵长类动物中可使循环 PCSK9 降低近 90%，血液 LDL-C 降低超过 60%，单次疗程后至少 8 个月效果持久。临床前概念验证研究阐明了基因组编辑方法治疗高脂血症的卓越治疗潜力，从而证实了它们向临床研究的进展。
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