The Angelman Approach : hacking DNA to treat a rare disease

Abstract

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.