In an article featured in Science Now, researchers reported at the annual meeting of the American Society of Gene Therapy that they could cure phenylketonuria in mice using a novel form of gene therapy. Phenylketonuria is a deficiency in phenylalanine hydroxylase that normally converts phenylalanine to tyrosine. The disease results in buildup of phenylalanine in the brain, leading to mental retardation.
Gene therapy has previously proven to be a problem because of the random integration of viral DNA into the host genome, possibly leading to disease. Recently, 3 patients that underwent gene therapy later developed leukemia.
The novelty of this new gene therapy is the use of specific localizing integration into the genome through the use of Integrase. This work has been pioneered by Michele Calos‘s group at Stanford. This approach alleviates random integration of the therapeutic DNA and possible disruption of essential genes.