Genome Editing of PCSK9 In Vivo (p 488)
With a single injection Ding et al reduce cholesterol levels in mice.
High levels of low-density lipoprotein (LDL) in the blood are one of the primary risk factors for coronary heart disease, and even though statins can decrease LDL levels, their use does not completely mitigate the risk of heart disease. Moreover, some patients are intolerant of statin therapy. Hence, new strategies for reducing LDL levels are highly desirable. In humans, LDL levels are regulated by the liver enzyme PCSK9, and individuals with loss-of-function mutation in the PCSK9 gene are healthy but have lower LDL levels and a lower risk of coronary heart disease. Ding and colleagues therefore wondered whether mutating the Pcsk9 gene artificially in mice would mimic the benefits seen in human PCSK9 mutants. To mutate Pcsk9, …