In this week's episode we explore gene therapies. We look at what genes are, why mutations can be dangerous, and what methods are being investigated to solve these problems. Then we focus on familial hypercholesterolemia as an example and look at what's being researched today to solve this problem.

References:

• Hoekstra M, Van Eck M. Gene editing for the treatment of hypercholesterolemia. Current Atherosclerosis Reports. 2024 May;26(5):139-46. https://doi.org/verve10.1007/s11883-024-01198-3
• Stankov S, Cuchel M. Gene editing for dyslipidemias: New tools to “cut” lipids. Atherosclerosis. 2023 Mar 1;368:14-24. https://doi.org/10.1016/j.atherosclerosis.2023.01.010
• Centers for Disease Control and Prevention. Cardiovascular disease. Centers for Disease Control and Prevention website. Published June 3, 2024. Accessed March 12, 2026. https://www.cdc.gov/cdi/indicator-definitions/cardiovascular-disease.html
• Canepari C, Cantore A. Gene transfer and genome editing for familial hypercholesterolemia. Frontiers in Molecular Medicine. 2023 Apr 3;3:1140997. https://doi.org/10.3389/fmmed.2023.1140997
• Zhao H, Li Y, He L, Pu W, Yu W, Li Y, Wu YT, Xu C, Wei Y, Ding Q, Song BL. In vivo AAV-CRISPR/Cas9–mediated gene editing ameliorates atherosclerosis in familial hypercholesterolemia. Circulation. 2020 Jan 7;141(1):67-79. https://doi.org/10.1161/CIRCULATIONAHA.119.042476
• Horie T, Ono K. VERVE-101: a promising CRISPR-based gene editing therapy that reduces LDL-C and PCSK9 levels in HeFH patients. https://doi.org/10.1093/ehjcvp/pvad103

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