Is there a gene therapy for phenylketonuria?
Strict phenylalanine-free and high tyrosine diet is still the current treatment for phenylketonuria. Foods that contain phenylalanine like meat, egg, fish, and the like are not given to affected children so as to avoid the potential damage caused by elevated phenylalanine levels in the blood. Foods that contain high amounts of tyrosine are given because the amino acid is the precursor for many physiologically important hormones and neurotransmitters. Furthermore, foods that contain low amounts of phenylalanine are specially processed to remove the amino acid for consumption by phenylketonurics. In 2005, Drs. Woo and Eisensmith of the USA reported a successful phenylketonuria gene therapy in laboratory mice. “Gene therapy is the delivery of genetic material to specific cell types of an organism to alter its physiology or function.” (Woo and Eisensmith 2005) The two biomedical scientists inserted the phenylalanine hydroxylase gene into the chromosomes of mouse hepatic cells via a bacteri