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Genetically Editing Out Human Disease

Tue, 01/19/2010 - 10:53am
Duke University

Many of the world’s most devastating diseases – such as hemophilia, cystic fibrosis and muscular dystrophy – are the result of a single gene defect, or mutation. If the Encyclopedia Britannica represented the human genome, such diseases would be analogous to one typographical error rendering the massive tome unreadable. The daunting task then is to find and fix the typo to make the encyclopedia readable again.

Charlie Gersbach is one of these genetic editors. In the biochemical equivalent of “cut and paste,” he is developing a series of genetic manipulations to replace the genetic “typo” with a functional one.

Because of his early successes in the field, Gersbach was recruited to the faculty of Pratt School of Engineering’s Biomedical Engineering Department as assistant professor, after a post-doctoral fellowship at the Scripps Research Institute in La Jolla, Calif.

But this foray into the complex world of genetics might have been derailed early if it wasn’t for a common household product.

While wrapping up his undergraduate degree in chemical engineering at the Georgia Institute of Technology, he applied for a prestigious internship at consumer giant Kimberly-Clark. After a grueling and competitive process, he finally landed the coveted spot. Shortly before he reported to work, however, he was informed that he was assigned to help develop the company’s latest breakthrough consumer product – moist toilet paper.

He quickly started looking in another direction. So he stayed in Atlanta and spent the next five years as a graduate research assistant at Georgia Tech and Emory University, earning a Ph.D. in biomedical engineering. It was here he also started tinkering with genetics.

Now, instead of having to explain something potentially embarrassing, though simple, to friends and family when asked what he does for a living, he now speaks in a language that only a handful of specialized researchers truly understand.

In many ways, a gene is like a computer program – it tells the cell what to do, whether to produce a protein or to guide a cell’s division. What Gersbach’s lab is developing is the ability to peer into the program to see the actual code that runs it. Once the malfunctioning code has been located, he has come up with a way to replace it with correct code, allowing the cells to perform their normal functions.

“What we are trying to do is control cell behavior,” Gersbach said. “For example, while at Georgia Tech we came up with a method to transform muscle cells into bone cells. This could have important implications in the finding new approaches to treating orthopedic diseases.”

Gersbach continued this line of research at Scripps. He had always wanted to go to Southern California, but after more than two years of west coast living, the southern New Jersey native grew homesick for the East Coast.

“My lab bench at Scripps literally overlooked the ninth hole of the Torrey Pines Golf Course, where they’ve played the U.S. Open,” he said. “I could sit there and watch the big names – including Tiger Woods – as they played the hole. Beyond the green, I could also see the spouts of migrating whales traveling up and down the coast. It was quite a view.”

While his primary appointment is in Pratt’s biomedical engineering department, his lab is located among other researchers in the surgery department’s Division of Surgical Services, whose mission is to develop novel molecular therapeutics. This location in a medical, non-engineering building is a concrete manifestation of how he straddles the engineering and medical worlds.

“The location so close to a medical school is such a great benefit for us bioengineers,” Gersbach said. “At Georgia Tech, we did not have such close interactions, since Emory was located across town. Another advantage is that the biomedical engineering department here attracts some of the best students around. It’s these types of highly motivated students who do a lot of the great work. I am looking forward to teaching them and being a mentor.”

In fact, within one month of starting in August, 2009, he already filled his allotment of three graduate students, who are each working on different aspects of Gersbach’s genetic research.

When Gersbach talks about his day at work with his wife Catherine, he has someone who understands the language. A noted biomaterials researcher in her own right, she has researched coatings that help artificial joint implants resist infections and wear. She is currently at home with their first-born.

Even though he is in a new town, in a new job, and with a new baby, the most important question facing Gersbach is: When will his passion for the Georgia Tech Yellow Jackets cool and warm to the Blue Devils?

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