Repair of DNA by gene editing techniques might be the way forward to treat metabolic diseases in the future
Genetic metabolic disease cured in mice by gene editing - health science news

By using a new genome editing technique, researchers have repaired a gene (phenylalanine hydroxylase) in the liver that causes the metabolic disease known as phenylketonuria. This disease should be diagnosed as soon as possible in babies, because the baby’s diet has to be phenylalanine-free to prevent accumulation of this amino acid, and subsequent mental retardation. Gene repair was achieved by delivering the genes necessary for the repair in a harmless virus that infects liver cells. These cells will make use of the genes brought by the virus, and repair the gene, so that the liver can metabolize phenylalanine. Thus, this new gene editing technique holds great promise for the future treatment of this and other metabolic diseases in humans.

Read the full story: ETH Zürich
Scientific publication: Nature Medicine