Because the liver is the site of many vital functions, impairment of only one protein within a complex metabolic pathway is usually highly deleterious. Such a condition is called inborn error of metabolism, and concerns many genetic diseases linked to a non-functional enzyme in the liver. Treatments, and longterm management, are currently not efficient enough, and patients would greatly benefit from an innovative therapy that meets this medical need. Orthotopic liver transplantation is the only radical treatment of severe defects, and/or end stage diseases. Cell therapy has been identified as the best alternative tool to overcome scarcity of organ donation. Several cell types are under investigation, and adult liver stem/progenitor cells represent an attractive cell source for liver regenerative medicine.
Promethera summer 2010