Curing Disease Through Genome Editing
An innovative gene therapy technique, known as genome editing, ‘searches’ a specific mutated gene and fixes it. Now, for the first time, it has been shown to work in living animals. In the study, the researchers used two versions of a genetically engineered virus (AAV, or adeno-associated virus). One of these versions carried the enzyme that cut the DNA in the right spot, and the other one transported the gene meant to replace the mutated one. All this was done in liver cells of living mice that suffered from the blood-clotting disorder hemophilia, which is caused by a single-gene mutation and comes in two forms: hemophilia A and hemophilia B, caused by, respectively a lack of clotting factor VIII and IX. In this study, the mice suffered from the B variant. read more
