Is Lifelong LDL-C Lowering Within Reach? The heart-1 Gene-Editing Trial

This transcript has been edited for clarity.

Michelle L. O'Donoghue, MD, MPH: Hi. This is Dr Michelle O'Donoghue, reporting for Medscape. I'm here at the American Heart Association meeting, and one of the interesting stories that we're going to be talking about is that of gene editing.

Joining me to discuss this is Dr Sekar Kathiresan. He's the co-founder and the chief executive officer of Verve, and they presented some very interesting results. Before we get into the results, let's take a step back and talk about the field in general. There are many people who may not be familiar with gene editing, so maybe you can walk us through a little bit of the basics.

CRISPR Scissors vs Eraser

Sekar Kathiresan, MD: Michelle, thank you so much for having me. As you know, the gene-editing technology was initially described in 2012 by Emmanuelle Charpentier and Jennifer Doudna, and the first generation of this technology, or so-called genetic scissors — that's really the analogy — is CRISPR-Cas9.

How CRISPR-Cas9 worksis, when given a guide RNA, it goes to a specific place in the genome based on that address that you gave in the guide and then directs a cut. It basically cuts the DNA entirely at that intended spot and has been used to basically turn off genes.