Gene editing disrupts Huntington's mutation in mice

Gene editing disrupts Huntington's mutation in mice

Researchers have developed a way to edit the genetic sequences at the root of Huntington's disease and Friedreich's ataxia. If longer than a certain threshold length, these sequences grow in length uncontrollably and lead to brain cell death in Huntington's disease, and the breakdown of nerve fibers in Friedreich's ataxia. There are no treatments that stop the progression of these diseases. Using base editing, the team introduced single-letter c…