By using bacterial proteins to edit genes in mice, scientists have reversed a rare liver disorder in the animal.
The disorder, called type I tyrosinemia, affects about one in 100,000 people and is caused by a single genetic mutation. In the study, scientists used a technique called CRISPR to remove the mutated DNA and insert the correct gene into some of the mice’s liver cells. The healthy cells reproduced and replaced the diseased cells, allowing the mice to survive without medication, the researchers report March 30 in Nature Biotechnology.
