Molecular Therapy Family of Journals (@moltherapy.bsky.social)

In vivo gene editing for lipid management: An early perspective from ANGPTL3-targeted CRISPR-Cas9 therapy Lifelong exposure to elevated levels of atherogenic lipoproteins increases the likelihood of developing atherosclerotic cardiovascular disease (ASCVD). Contemporary lipid-lowering pharmacotherapies have transformed cardiovascular prevention; however, their effectiveness is constrained by the need for lifelong treatment, highly variable adherence, and persistent residual cardiovascular risk. Human genetic studies of naturally occurring loss-of-function variants in genes regulating lipid metabolism, including ANGPTL3, demonstrate that suppression of these pathways is associated with markedly lower levels of atherogenic lipoproteins and a substantially reduced lifetime risk of ASCVD with no apparent harmful effects. http://dlvr.it/TQ7DlC