Select your localized edition:

Close ×

More Ways to Connect

Discover one of our 28 local entrepreneurial communities »

Be the first to know as we launch in new countries and markets around the globe.

Interested in bringing MIT Technology Review to your local market?

MIT Technology ReviewMIT Technology Review - logo

 

Unsupported browser: Your browser does not meet modern web standards. See how it scores »

For the PNAS study, the Alnylam researchers designed short, double-stranded RNA molecules to silence the gene for PCSK9 in rodents, monkeys, and humans. They packaged the molecules into lipid-based nanoparticles developed by biomedical engineer Robert Langer and his group at MIT. The nanoparticles protect the molecules in the bloodstream and escort them to liver cells.

Injecting the drug into mice and rats lowered total cholesterol by up to 60 percent, and in monkeys, a single dose cut LDL cholesterol by 50 to 60 percent. The reduction lasted about three weeks. Although PCSK9’s importance was clear from genetic studies in rodents and humans, “what was not known was, if you were to acutely knock down the level of PCSK9, how long would it take for cholesterol to go down,” Fitzgerald says. “The answer was, if you knock it down today, then your cholesterol is down tomorrow.”

It’s not yet clear how well an RNA-interference-based drug that requires injections could compete with existing medicines for lowering cholesterol. No such drugs have yet been approved by the Food and Drug Administration, although several are in clinical trials. While there have been some safety concerns with RNA-based therapeutics, scientists at Alnylam say that they saw no unacceptable side effects in animals given the cholesterol-lowering treatment, and people who naturally lack PCSK9 seem healthy.

3 comments. Share your thoughts »

Credit: Alnylam Pharmaceuticals

Tagged: Biomedicine, Alnylam, cholesterol, gene silencing, RNA interference

Reprints and Permissions | Send feedback to the editor

From the Archives

Close

Introducing MIT Technology Review Insider.

Already a Magazine subscriber?

You're automatically an Insider. It's easy to activate or upgrade your account.

Activate Your Account

Become an Insider

It's the new way to subscribe. Get even more of the tech news, research, and discoveries you crave.

Sign Up

Learn More

Find out why MIT Technology Review Insider is for you and explore your options.

Show Me
×

A Place of Inspiration

Understand the technologies that are changing business and driving the new global economy.

September 23-25, 2014
Register »