Key result
Gene therapy, including RNA-targeted therapies and gene editing technologies, shows excellent lipid-lowering promise for patients with familial hypercholesterolemia.
Why the study?
Existing lipid-lowering therapies struggle to achieve target lipid levels in patients with familial hypercholesterolemia, especially those with homozygous disease.
Does gene therapy improve lipid-lowering in patients with familial hypercholesterolemia?
Does gene therapy improve lipid-lowering in patients with familial hypercholesterolemia?
Gene therapy, including RNA-targeted therapies and gene editing, shows excellent promise for achieving lipid targets in patients with familial hypercholesterolemia.
Gene therapy advances may address refractory homozygous FH; leaves open safety, durability, and efficacy in prospective trials.
(1) Background: Existing lipid-lowering therapies have difficulty in achieving lipid target levels in patients with familial hypercholesterolemia (FH), especially in the treatment of patients with homozygous familial hypercholesterolemia. (2) Method: All of the literature data containing "Familial hypercholesterolemia" and "Gene Therapy" in PubMed and Clinical Trials from 2018 to 2022 were selected. (3) Results: The rapid development of gene therapy technology in recent years is expected to change the treatment status of FH patients. As emerging gene therapy vectors, the optimized adeno-associated viruses, exosomes, and lipid nanoparticles have demonstrated an improved safety and higher transfection efficiency. Various RNA-targeted therapies are in phase 1-3 clinical trials, such as small interfering RNA-based drugs inclisiran, ARO-ANG3, ARO-APOC3, olpasiran, SLN360, and antisense oligonucleotide-based drugs AZD8233, vupanorsen, volanesorsen, IONIS-APO(a)Rx, etc., all of which have demonstrated excellent lipid-lowering effects. With gene editing technologies, such as CRISPR-Cas 9 and meganuclease, completing animal experiments in mice or cynomolgus monkeys and demonstrating lasting lipid-lowering effects, patients with FH are expected to reach a permanent cure in the future. (4) Conclusion: Gene therapy is being widely used for the lipid-lowering treatment of FH patients and has shown excellent therapeutic promise, but the current delivery efficiency, economic burden, immunogenicity and the precision of gene therapy can be further optimized.
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Fu et al. (2022) conducted a review in Familial hypercholesterolemia. Gene therapy was evaluated. Gene therapy, including RNA-targeted therapies and gene editing technologies, shows excellent lipid-lowering promise for patients with familial hypercholesterolemia.
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