Pig Model May Aid Work on PKU Treatments, Including Gene Therapies

Pig Model May Aid Work on PKU Treatments, Including Gene Therapies

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Pig Model May Aid Work on PKU Treatments, Including Gene Therapies

A first-of-its-kind pig model of phenylketonuria (PKU) has been developed that could be useful in advancing potential disease treatments, including gene therapies. The model was described in the study “Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing,” published in PLOS One. PKU is caused by mutations in the PAH gene, which encodes an enzyme of the same name that is necessary to break down an amino acid called phenylalanine. The lack…

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