Why the study?
Many therapies for dyslipidemia are limited by suboptimal long-term adherence, prompting exploration of gene editing and epigenetic therapies.
Do gene editing therapies reduce lipid and lipoprotein risk factors in patients with dyslipidemia?
Population
Patients with dyslipidemia and atherosclerotic cardiovascular disease risk factors
Comparison
Gene editing, base editing, and epigenomic editing therapies targeting ANGPTL3 and PCSK9
Design
Narrative review
Key result
Gene editing and epigenetic therapies for dyslipidemia demonstrate favorable reductions in ANGPTL3, PCSK9, LDL cholesterol, and triglycerides in early phase clinical evaluation.
Authors
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Captured external expert commentary on this paper, strongest first. Original sources are linked where available.
“These data, although early, really are appropriate to allow further development. But thinking about a one-time therapy that can lower LDL cholesterol by about 50% and triglycerides greater than 50% is exciting. It very well may overcome this issue of waning adherence to current [lipid-lowering] therapies.”
“The ability to safely edit an individual's genome to permanently reduce atherogenic lipoproteins could overcome the significant challenge of waning adherence to current lipid-lowering therapies that require chronic administration. Although it is early in the study of gene-editing therapies, the efficacy shown in this phase 1 trial is quite promising and represents a new frontier for drug development.”
“ongoing surveillance will be essential to assess the persistence of safety and lipid-lowering efficacy of CTX310.”
Should not yet change dyslipidemia practice; leaves open gene editing's role pending larger trials.
Do gene editing therapies reduce lipid and lipoprotein risk factors in patients with dyslipidemia?
Gene editing therapies targeting ANGPTL3 and PCSK9 show early promise for robust and durable reductions in atherogenic lipids, though further clinical trials are needed.
Stephen J. Nicholls (2026) conducted a review in dyslipidemia. Gene editing and epigenetic therapy was evaluated. Gene editing and epigenetic therapies for dyslipidemia demonstrate favorable reductions in ANGPTL3, PCSK9, LDL cholesterol, and triglycerides in early phase clinical evaluation.