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Therapeutic application of genome editing in dyslipidemia
Madelynn N Whittaker1, Kiran Musunuru2
1Department of Bioengineering, School of Engineering and Applied Science.
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.
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