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Mohammadsharif Tabebordbar

Broad Institute of MIT and Harvard

When Sharif Tabebordbar was a teenager in Iran, he watched his father slowly lose the ability to walk. The cause was a form of muscular dystrophy.  “He resisted it for a long time. He refused to sit in a wheelchair. But there was a point when he just could not walk on his own anymore,” Tabebordar said. “He delayed it as much as he could.”

Tabebordbar was in his early 20s at the time, studying in Tehran. When he started looking into doing a Ph.D. in the U.S., he only applied to schools that had active research on diseases like his father’s. One of the most promising ways to treat this sort of illness is to use a harmless virus as a messenger, to deliver a functional copy of whatever gene isn’t working properly. But it can be hard to direct the virus to get into the muscle itself; often, much of it gets sequestered in the liver.

What Tabeborbdar did was engineer millions upon millions of variants of that virus in a single test tube, and then inject all of them into a mouse or monkey. By sequencing the animal’s muscle tissue, he could tell which variant got into that tissue the best. The technique was promising enough for him to start a company called Kate Therapeutics. “Kate is the name of a patient with a genetic muscle disease,” Tabebordbar explained. His hope is to use his million-variants method to find a safe and effective treatments and move into clinical trials as soon as possible.

Eric Boodman