High cholesterol lowered with single in vivo gene edit

What's the news: Heart disease patients had their cholesterol levels permanently lowered with a single gene editing treatment.

Why should we believe it: This news is based on an announcement made yesterday at the American Heart Association Scientific Sessions by scientists from Verve Therapeutics. The results come from a small but exciting study, involving 10 chronic heart disease patients.

The patients each received a CRISPR-based gene therapy to change a single gene in the liver. The gene edit mimicked a natural genetic variant that protects against heart disease. Results:

• Dose-dependent reductions of between 40%-55% in LDL cholesterol.

• Effects lasting 6+ months.

• Mild and transient side effects ("flu-like symptoms for a few hours after infusion").

Why this is a big deal: Nearly 25 million Americans have high cholesterol (over 240 mg/DL). A large number of those with high cholesterol are not taking medication and are at increased risk of heart disease. In other words, the current intervention could help millions of people with a single treatment.

Beyond the direct impact, this research points to a future where we might make safe, easy, simple tweaks to our own DNA to cope with various diseases and conditions. THis might not be just heart disease, but neurological conditions, cancer, or even simple old age.

So what's next: Verve has partnered with Eli Lilly and will roll out larger trials of its treatment soon. Stay tuned to Long Youthspan, because I will have more updates on this exciting new direction in medical research.