As reported in EurekAlert!, Children’s Hospital Los Angeles Investigator Tracy Grikscheit MD has received $1.3 million to study the use of pluripotent stem cells on liver-based metabolic disease. The grant was awarded by the California Institute of Regenerative Medicine (CIRM) to study stem cell therapy for liver failure. Grikscheit’s CIRM-funded project, Universal Pluripotent Stem Cell Therapy (UPLiFT), will treat liver failure resulting from metabolic diseases that normally are fatal without a liver transplant. These diseases include urea cycle disorders, the result of a genetic mutation that interferes with removal of toxic ammonia from the body, and Crigler Najjar syndrome, an inherited condition characterized by high levels of bilirubin, a toxic substance produced when red blood cells break down. Both of these disorders can be severe, causing brain damage, coma or death.
Tracy Grikscheit, MD