Researchers healed mice with a genetic metabolic disorder that also affects humans by using a new editing tool to target and correct genetic mutations.
Some babies are born with the metabolic disorder phenylketonuria and need a special diet so that the amino acid phenylalanine doesn’t accumulate in the body. Excess phenylalanine delays mental and motor development. If left untreated, the children may develop mental disabilities.
The cause of this metabolic disorder is a mutation in a gene that provides the blueprint for the enzyme phenylalanine hydroxylase (Pah). The enzyme, which is produced by the cells of the liver, metabolizes phenylalanine.
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