Therapeutic liver repopulation for phenylketonuria

  title={Therapeutic liver repopulation for phenylketonuria},
  author={Cary O. Harding and K. Michael Gibson},
  journal={Journal of Inherited Metabolic Disease},
Problems with long-term dietary compliance in phenylketonuria (PKU) necessitate the development of alternative treatment approaches. Therapeutic liver repopulation with phenylalanine hydroxylase (PAH)-expressing cells following hepatocyte or haematopoietic stem cell transplantation has been investigated as a possible novel treatment approach for PKU. Successful therapeutic liver repopulation requires both a stimulus for liver regeneration at the time of cell transplantation and a selective… CONTINUE READING