Scientists are testing gene therapy using CRISPR-Cas9 technology to treat high cholesterol, which targets the ANGPTL3 gene responsible for regulating LDL cholesterol and triglycerides[1][2][3]. A pilot study published in the New England Journal of Medicine showed a nearly 50 percent reduction in "bad" LDL cholesterol levels in patients with difficult-to-treat lipid disorders[1][2][3][5]. The therapy was administered as a single infusion to nine volunteers at various doses and was performed safely[3][4]. The inspiration was a natural genetic mutation of the ANGPTL3 gene, which occurs in one in 250 people and leads to lifelong low blood fat levels without negative effects[2][3]. This mutation reduces the risk of cardiovascular diseases[2][3]. The study is at a very early stage with a small number of participants and requires further research to verify safety and efficacy[1][2]. In the future, the therapy could replace the long-term use of drugs such as statins[2][3].