Therapeutic application of genome editing in dyslipidemia.

ABSTRACT

PURPOSE OF REVIEW To summarize recent advances with respect to the use of genome editing to modify blood lipid levels in vivo. RECENT FINDINGS: Genome-editing technologies have been successfully used to target the PCSK9 gene in the livers of nonhuman primates and significantly reduce blood LDL cholesterol levels. SUMMARY: Multiple proof-of-concept nonhuman primate studies raise the prospect of genome editing empowering 'one-and-done' therapies for the treatment of dyslipidemic patients.