According to a story from The Telegraph, a team of Chinese and American scientists have used gene editing to create pigs with Huntington’s disease. A portion of human genetic material that contains the mutation responsible for the disease was introduced to the pig DNA. The researchers are hoping to use the pigs for medical testing in order achieve a new breakthrough in treating this debilitating and deadly disease.
Huntington’s disease is a genetic disorder that causes the cells of the brain to die. While symptoms can start at any age, they usually begin in middle age, anywhere from 30 to 50 years. They often begin subtly, starting with changes to personality and behavior. This is followed by progressively worsening loss of coordination and abnormal gait. As the disorder progresses, symptoms include jerking movements called chorea, loss of walking ability, problems with talking, chewing, and speaking, and dementia. Most people die from the disease 15 to 20 years after getting diagnosed. There is no cure available, although some treatments can help reduce the severity of symptoms. To learn more about Huntington’s disease, click here.
Typically, mice are the most common animal model used for medical testing, and in general they work as a fairly consistent model for human diseases. However, in Huntington’s disease, the story is different. Testing Huntington’s disease in mice does not work well because mice do not experience the same symptomatic profile as humans. Because of this, many tests and treatments that appeared to work well for mice were not as effective in human trials. This is why the research team decided to transplant the disease to pigs as an alternative.