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 »

Frank Giordano’s patients need new blood vessels: some have blocked arteries around their hearts, and others have poor circulation in their limbs. In a few cases, the Yale University cardiologist and molecular biologist has coaxed new vessels to grow using conventional gene therapy, injecting patients with genes that encode a natural protein called vascular endothelial growth factor. But he’s most excited about a new treatment that, instead of relying on imported genes, stimulates the patients’ own genes to work harder.

For now, Giordano’s patients are strictly of the furry rodent variety, but the substances he’s testing-engineered transcription factors that act directly on DNA to stimulate or block protein production-could herald a new approach to treating human disease. Sangamo BioSciences in Richmond, CA, designs transcription factors that bind uniquely to DNA sequences near specific medically relevant genes and turn protein production up or down at the source. “It’s like a rheostat for any protein you want,” says Casey Case, Sangamo’s vice president of research.

Case says the technique has distinct advantages over existing gene therapy methods. In gene therapy-still an experimental treatment itself-genes are injected into a patient and provide instructions for making a single version of a protein; but native genes often code for several versions of a protein, each of which can play an important biological role. Because an engineered transcription factor turns on the body’s own copy of a gene, all of these versions of a protein are made. Giordano says, for example, that mice producing all the variants of the blood vessel growth factor seem to have “more robust” vessels. And because Sangamo can engineer transcription factors for any genes-including those involved in cancer, diabetes, heart disease or nerve repair-“the sky’s the limit” for other therapeutic applications, he says.

Paul Robbins, a gene therapist at the University of Pittsburgh, speculates that the approach could also be used in treatments for cancer and inflammation-and he predicts that with further research, the technique could offer even more control over gene expression. “This technology is at its very beginning,” Robbins says.

Right now, an engineered transcription factor can reach a patient’s DNA only if a gene encoding the factor is delivered to the relevant cells via gene therapy, but Sangamo is working to synthesize transcription factors that could be delivered intravenously. The company hopes to be ready for human trials of its vascular endothelial growth factor stimulator by late 2003, and if they pan out, cardiologists might have a way to help less furry patients build healthier hearts.

1 comment. Share your thoughts »

Tagged: Biomedicine

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 »