n=15, but this is still an impressive example of gene therapy, especially since it doesn't addresses a rare single-gene disease, but the usual case of a much more complex genetic and metabolic system.
>15 patients received a one-time infusion of lipid nanoparticles, essentially fat bubbles, carrying gene-editing machinery to their livers. That machinery was programmed to snip and disable a gene involved in cholesterol metabolism called ANGPTL3.
>The experiment was inspired, in part, by the discovery that there are people who have naturally occurring versions of the ANGPTL3 gene that aren’t functional. Those people have very low levels of cholesterol and triglycerides and a lower risk of cardiovascular disease.
marojejian•1h ago
n=15, but this is still an impressive example of gene therapy, especially since it doesn't addresses a rare single-gene disease, but the usual case of a much more complex genetic and metabolic system.
>15 patients received a one-time infusion of lipid nanoparticles, essentially fat bubbles, carrying gene-editing machinery to their livers. That machinery was programmed to snip and disable a gene involved in cholesterol metabolism called ANGPTL3.
>The experiment was inspired, in part, by the discovery that there are people who have naturally occurring versions of the ANGPTL3 gene that aren’t functional. Those people have very low levels of cholesterol and triglycerides and a lower risk of cardiovascular disease.