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Phenylketonuria (PKU) is one of the most prevalent inherited metabolic diseases and is accountable for a severe encephalopathy by progressive intoxication of the brain by phenylalanine. This results from an ineffective L-phenylalanine hydroxylase enzyme (PAH) due to a mutated phenylalanine hydroxylase ( PAH ) gene. Neonatal screening programs allow an early dietetic treatment with restrictive phenylalanine intake. This diet prevents most of the neuropsychological disabilities but remains challenging for lifelong compliance. Adult-derived human liver progenitor cells (ADHLPC) are a pool of precursors that can differentiate into hepatocytes. We aim to study  PAH  expression and  PAH  activity in a differenciated ADHLPC. ADHLPC were isolated from human hepatocyte primary culture of two different donors and differenciated under specific culture conditions. We demonstrated the high expression of  PAH  and a large increase of  PAH  activity in differenciated LPC. The age of the donor, the cellular viability after liver digestion and cryopreservation affects  PAH  activity. ADHLPC might therefore be considered as a suitable source for cell therapy in PKU.

Adult human liver mesenchymal progenitor cells express phenylalanine hydroxylase