Weekly reads: effective LDL gene editing, Chinese genetics guidelines, human embryo models in space

If you take a statin to improve your lipid profile, someday could you undergo LDL gene editing instead? Might that day be sooner than we think? A new paper has generated great interest in this topic including in the media.

LDL gene editing

For example, we have: One-and-Done Heart Disease Prevention? Scientists Show It May Be Possible. A single infusion of an experimental gene-editing drug seemed to reduce LDL long-term in a small trial. The results may point to something “curative,” one expert said.NYT. “The study was led by Dr. Sekar Kathiresan, the chief executive at Verve Therapeutics, now a subsidiary of Eli Lilly. Dr. Kathiresan, a cardiologist, said he was motivated by personal history.”

Here’s the NEJM paper: In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia. The long-term safety of such LDL gene editing is unknown, but it’s an intriguing approach. Can “one-and-done” gene editing potentially make taking pills from some conditions unnecessary? There’s a long road to that point.