Stem Cells, Vol 13, 613-621, Copyright © 1995 by AlphaMed Press
The use of umbilical cord blood as a cellular source for correction of genetic diseases affecting the hematopoietic system
DW Clapp and DA Williams
Herman B. Wells Center for Pediatric Research, Department of Pediatrics, Indianapolis, Indiana 46202-5210, USA.
Human umbilical cord blood contains abundant primitive and committed
hematopoietic progenitors and has been used as an alternative source of
reconstituting hematopoietic stem cells. Recent advances in the
understanding of molecular aspects of multiple diseases and improvements in
technology associated with prenatal diagnosis now allow the in utero
identification of many genetic diseases affecting the hematopoietic system.
Advances in technology raise the potential for genetic correction and
subsequent transplantation of autologous cord and placental blood
hematopoietic stem cells into affected patients prior to expression of the
disease phenotype. This review will summarize the recent data on advances
in prenatal diagnosis, characterization of the biology of cord blood stem
cells, and efforts at developing methods for genetic transduction of cord
blood hematopoietic stem/progenitor cells.
