Purpose of review: The purpose is to review recent progress in applying the CRISPR/Cas9 system to lipid metabolism and therapeutics.
Recent findings: The CRISPR/Cas9 system has been used to generate knockout animals for lipid genes in multiple species. Somatic genome editing with CRISPR/Cas9 can efficiently disrupt genes in adult animals, including a new strategy for generating atherosclerosis. Refinements to the CRISPR/Cas9 system including epigenetic modulators and base editors offer new avenues to manipulate gene expression. The recent report of germline genome editing in humans highlights the promise as well as perils of this technology.
Summary: CRISPR/Cas9 is a transformative technology that will help advance on our understanding of lipid metabolism and physiology. Somatic genome editing is a particularly promising approach for editing genes in tissues of live organisms, and represents a new means of addressing unmet therapeutic challenges in humans. Educational outreach, public debate, and consideration of ethics and safety must guide the use of genome editing in humans.