Therapeutic application of genome editing in dyslipidemia
Madelynn N. Whittaker, Kiran Musunuru
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.
Topics & Concepts
Genome editingDyslipidemiaComputational biologyBiologyGenomeBioinformaticsMedicineGeneticsGenomicsHuman genomeMEDLINELipoproteins and Cardiovascular HealthCRISPR and Genetic EngineeringVirus-based gene therapy research