In Vivo Gene Editing Lowers Cholesterol Dramatically

Statins and PCSK9 inhibitors can dramatically lower LDL cholesterol levels and improve cardiovascular outcomes — if patients take the pills or receive the injections regularly for many years. In a biotechnology industry–supported study, researchers sought to determine if a one-time gene therapy treatment might achieve the same goals. The team created a lipid nanoparticle that would be taken up selectively by hepatocytes (where the PCSK9 protein is produced). Inside the nanoparticle was a gene-editing complex — a base editor — that substitutes an inosine for an adenosine at a specific spot in the PCSK9 gene, which greatly reduces the production of the PCSK9 protein. After several years of encouraging experiments in animals to assess e…