Stem cell therapy for rare liver metabolic disorders
A subset of rare congenital metabolic liver diseases is amenable to treatment by the replacement of faulty liver cells. While cellular transplants have been attempted in patients, these have failed because the donor cells did nor persist or were immune rejected. Additionally, it is very difficult to find a donor liver from which to isolate cells for transplantation. We propose a new strategy to grow large numbers of liver progenitor cells from a patient so that these could be genetically corrected in the petri dish and reintroduced back into the same patient. These type of personalized stem cell therapy has the potential to produce long-lasting cures for these disorders.