Treatment of Rare Genetic Disorders with Umbilical Cord Blood Transplantation: Hints about Tissue Repair and Regeneration

Joanne Kurtzberg
Department of Pediatrics - Bone Marrow Program; Duke University Medical Center

Cord blood stem and progenitor cells can rescue the bone marrow and immune system of patients undergoing myeloablative chemotherapy. Cord blood cells may also be capable of transdifferentiation into non-hematopoietic lineages a property that would make them ideal candidates for cell therapy to induce tissue regeneration and repair. Since 1995, we have transplanted >150 infants and children with inborn errors of metabolism. Post transplant, we have seen engraftment of donor cells in damaged tissues including the heart, liver, brain and blood vessels. Repair of bone, cartilage, corneas have also been seen. Transdifferentiation of cells into oligodendrocytes and cardiac myocytes has also been observed both in vivo and in vitro. Lessons learned from transplantation of patients with these rare diseases will lead to the development of novel cell therapies for more common adult degenerative diseases.