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 | From the Editor's Desk
If DNA is like software, can we just fix the code? When you first meet her, you won't be able to tell that Ipek Kuzu suffers from a rare genetic disease. The three-year-old plays happily on her own for hours, driving her toy cars and "cooking" in her pretend kitchen. But she's not well. She's a little wobbly on her feet and doesn't say much, and if nothing is done, she may die by her mid-20s. Ipek has ataxia-telangiectasia, or A-T, a disease caused by an error in her DNA. It causes the loss of brain cells, along with a high risk of infection and cancer.
It's the sort of problem that makes doctors shake their heads. But Ipek's father, Mehmet, and mother, Tugba, hope she'll escape that fate. Thanks in part to the persistence of Mehmet, a programmer at Google, in January she became one of the first handful of US patients to receive a hyper-personalized gene medicine, tailored to treat a unique mutation. The one-person drug, designed for her by a Boston doctor, Timothy Yu, is being called "atipeksen," for "A-T" and "Ipek."
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