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The Angelman Approach: Hacking DNA to Treat a Rare Disease

May 31, 2017

Recent advances are allowing clinicians to treat genetic illnesses by directly manipulating patients’ DNA, and a number of scientists are now investigating ways to leverage those discoveries for individuals with Angelman Syndrome. Their work could lead to potent therapies for the disease, and – maybe – even a cure.

One of every hundred children is born with a disease caused by a single abnormal gene. In the case of Angelman Syndrome, the genetic defect leaves patients mentally disabled, largely or completely unable to speak, and prone to seizures and sleep difficulties. Many Angelman researchers are trying to figure out precisely how those symptoms develop, but why study all the individual effects when you could go right to the root of the problem? Recent advances in medicine and technology are increasingly allowing clinicians to treat genetic illnesses by directly manipulating patients’ DNA, and a number of scientists are now investigating ways to leverage those discoveries for individuals with Angelman Syndrome. Their work could lead to potent therapies for the disease, and – maybe – even a cure.

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Tagged asAngelman Syndrome genetics health medicine

Written by

Brandon Levy

Brandon was born in Boston but raised down the street from the National Institutes of Health (NIH) in Chevy Chase, Maryland. After bursting onto the reporting scene with an investigation of the food served in his middle school’s cafeteria, Brandon went on to win several writing competitions and serve as editor-in-chief of his high school newspaper, The Tattler. Meanwhile, the combination of an early interest in biology and his family’s many eccentricities made him intensely curious about why people act the way they do. Brandon earned a B.S. in Neuroscience from Duke University, where he volunteered in a neuroimaging lab and wrote a senior thesis on the influence of emotional facial expressions on social decision making. After graduating, he returned to Maryland to work in the Laboratory of Brain and Cognition at the NIH’s National Institute of Mental Health. Finding the day-to-day grind of scientific inquiry to be less-than-thrilling, he began writing about NIH-funded research for several of the institution’s publications and was soon hooked. He has also worked as a member of the press team at the NIH’s National Institute of Neurological Disorders and Stroke, and now works with the NIH's Intramural Research Program (IRP) to help inform the public about the cutting-edge research being done at the NIH. When he’s not writing or reading Stephen King novels, Brandon enjoys singing, cooking, and cheering on Duke’s basketball team.

Thesis: The Angelman Approach: Hacking DNA to Treat a Rare Disease