Lipid Nanoparticles for Genome Editing in Hypercholesterolemia

Introduction: Hyperlipidemia is a medical condition that is characterized by elevated blood lipid levels. This condition is often associated with an increased risk of cardiovascular diseases, such as heart attack and stroke. Traditional treatment approaches for hyperlipidemia include lifestyle modifications, dietary changes, and pharmacotherapy. However, some patients cannot achieve the LDL-C treatment goals, necessitating the development of novel lipid-lowering therapies with improved efficacy and safety profiles. Gene editing, which permanently alters gene expression, can revolutionize disease treatment. The CRISPR/Cas9 system has emerged as the preferred technology among various gene editing approaches due to its high efficiency and relative simplicity. Nowadays, CRISPR-Cas9 technology is frequently used in medicine and many other fields for targeted gene screening and gene therapy. However, challenges remain in the clinical application of CRISPR-Cas9, and a safe and efficient delivery system is required. Method: The search was performed in Scopus, PubMed, and Google Scholar using the following keywords: “Hyperlipidemia,” “CRISPR-Cas9”, “Genome Editing,” “Gene Editing,” “Lipid Nanoparticles,” and “LNPs.” We included all relevant in vitro, in vivo, and clinical trial studies published until November 2024. Results: It has been indicated that the use of lipid nanoparticles (LNPs) as delivery vehicles is promising. The LNPs enhance the stability of the CRISPR/Cas9 system and offer advantages, such as ease of production and high versatility, making them an ideal carrier with great potential. Conclusion: This review provides an overview of the hopeful technology and the latest advances in the development of dyslipidemia treatments.