Clinical application of umbilical cord blood-derived stem cells for tissue regeneration

Abstract

Cord blood transplantation has been utilized as an alternate allogeneic stem cell source for more than two decades for the treatment of a variety of malignant and nonmalignant diseases, including hematologic malignancies (most prominently acute leukemias), marrow failure syndromes, hemoglobinopathies, and inherited lysosomal and peroxisomal storage diseases. Advantages of cord blood transplantation as compared to other allogeneic stem cell sources include greater HLA disparity without an increase in graft-versus-host disease (GVHD), faster availability and lower risk for somatic mutations and transmitting infections by latent viruses. Cord blood also contains multiple populations of stem cells and progenitors. In addition to hematopoietic stem and progenitor cells, non-hematopoietic stem cells with a variety of different regenerative potential have been identified in cord blood. The multitude composition and the plasticity of cord blood stem cells have elicited great interest in the therapeutic application of cord blood and its derived stem cells in tissue regeneration in non-hematopoietic organs. This chapter will discuss the development of cord blood transplantation as an autologous source for acquired diseases such as brain injury, and as a gene replacement therapy for inherited genetic diseases focusing on recessive dystrophic epidermolysis bullosa.