What genetics is good for

There’s an article in Nature News about one of the most interesting findings from large-scale genomic studies.  People with mutations in a gene called PCSK9 have low levels of cholesterol, and since the protein produced by that gene is active outside cells, it is relatively easy to target.  Also, people with mutations in both of their copies of the PCSK9 gene seem to be healthy, so it looks as though it should be a safe target for treatment

Synthetic antibodies against PCSK9 reduced LDL (bad) cholesterol by 73% in initial trials in a small group of patients, which is huge. There’s a huge trial going on at the moment to see if this translates to a reduction in heart attacks, strokes, etc.  It could still easily fail — several other drugs giving big cholesterol improvements have turned out not to prevent heart disease — but it is very promising.