Scientists often use a disabled virus called an adeno-associated virus, or AAV, to deliver gene therapy to cells. That includes muscular dystrophies that affect about 100,000 people in the United States, according to the Muscular Dystrophy Association.
His quest led him to a doctorate in developmental and regenerative biology, the most competitive ranks of academic medical research, and a discovery, published in September in the journal Cell, that could transform gene therapy - medicine that corrects genetic defects - for nearly all muscle wasting diseases. No one could answer the question plaguing Sharif and his younger brother, Shayan: What was this disease? And would they develop it the way their father had?Īs he grew up and watched his father gradually decline, Sharif vowed to solve the mystery and find a cure. Now he has to support one hand with another. Jafar Tabebordbar could walk when he was in his 30s but stumbled and often lost his balance. The mysterious illness would come to define Sharif’s life.
When Sharif Tabebordbar was born in 1986, his father, Jafar, was 32 and already had symptoms of a muscle wasting disease.
