allogeneic hepatocyte transplantation (Ira Fox)

Allogeneic hepatocyte transplantation is an experimental cellular therapy developed by Dr. Ira J. Fox at the Children's Hospital of Pittsburgh of UPMC for the treatment of classical phenylketonuria (PKU). The therapy involves the infusion of healthy donor-derived (allogeneic) primary human hepatocytes into the portal vein of the patient. These donor cells express functional phenylalanine hydroxylase (PAH), the enzyme responsible for converting phenylalanine to tyrosine, which is deficient in PKU patients. To enhance the engraftment and selective growth of the transplanted cells, patients undergo preparative intensity-modulated radiation therapy (IMRT) to a portion of the liver. The goal is to restore sufficient hepatic PAH activity to normalize blood phenylalanine levels and reduce the need for strict dietary restriction.

Other names

Hepatocyte Transplantation for Phenylketonuriaallogeneic hepatocytes