The use of umbilical cord blood as a cellular source for correction of genetic diseases affecting the hematopoietic system.
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.
Clapp
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Herman B. Wells Center for Pediatric Research, Department of Pediatrics, Indianapolis, Indiana 46202-5210, USA.
Williams
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DA
D000818:Animals; D005312:Fetal Blood / Q000166:cytology*; D018014:Gene Transfer Techniques; D006402:Hematologic Diseases / Q000235:genetics* / Q000473:pathology; D006410:Hematopoiesis / Q000502:physiology*; D006412:Hematopoietic Stem Cells / Q000502:physiology; D006801:Humans; D051379:Mice; D011296:Prenatal Diagnosis; D012190:Retroviridae / Q000235:genetics; D014161:Transduction, Genetic