Gene therapy and genome editing technologies, such as CRISPR base editing, offer potentially transformative single-course treatments for lipoprotein disorders by permanently inactivating cholesterol-raising genes.
Gene therapy and genome editing represent a promising frontier for the durable management of lipoprotein disorders, potentially revolutionizing ASCVD prevention by addressing the limitations of lifelong traditional lipid-lowering therapies.
Genetic factors play a critical role in the development of lipoprotein disorders, which significantly contribute to atherosclerotic cardiovascular disease (ASCVD). Traditional management of these conditions has relied on lipid-lowering therapies, which require lifelong adherence. Recent advancements in gene addition and editing technologies offer novel and potentially transformative approaches for treating lipoprotein disorders by targeting the relevant genetic pathways for each disease. This review revisits major monogenic and polygenic disorders of lipoprotein metabolism, including familial hypercholesterolemia, elevated lipoprotein(a), and familial chylomicronemia syndrome, and discusses the genetic-based therapies for management. RNA-based, gene addition and gene editing therapies, including Clustered Regularly Interspaced Short Palindromic Repeats, base editing and interventions whereby, are highlighted for their potential to provide durable treatments which overcome the adherence challenge. Integration of machine learning for risk prediction and the use of polygenic risk scores to enhance risk stratification further demonstrate the promise of personalized approaches, and overall potential for gene-based treatments to revolutionize ASCVD prevention and management.
Gurevitz et al. (Wed,) conducted a review in Lipoprotein disorders. Gene therapy and genome editing was evaluated. Gene therapy and genome editing technologies, such as CRISPR base editing, offer potentially transformative single-course treatments for lipoprotein disorders by permanently inactivating cholesterol-raising genes.