Cord blood transplantation and the potential for gene therapy. Gene transduction using a recombinant adeno-associated viral vector

Ann N Y Acad Sci. 1995 Dec 29:770:105-15. doi: 10.1111/j.1749-6632.1995.tb31048.x.

Abstract

Cord blood, which contains a high frequency of immature stem/progenitor cells with extensive proliferative and replating capacity in vitro was used as a clinical source of transplantable stem and progenitor cells. These cells can be efficiently transduced with new genetic material by using AAV or retroviral vectors. Using a recombinant AAV vector, high level expression of the lacZ gene under a CMV promoter was demonstrated in immature subsets of cord blood progenitor cells.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • DNA, Recombinant
  • Dependovirus / genetics*
  • Fetal Blood / cytology*
  • Gene Expression
  • Gene Transfer Techniques*
  • Genetic Therapy*
  • Genetic Vectors*
  • Granulocytes / metabolism
  • Hematopoietic Stem Cell Transplantation*
  • Hematopoietic Stem Cells / metabolism
  • Humans
  • Infant, Newborn
  • beta-Galactosidase / genetics

Substances

  • DNA, Recombinant
  • beta-Galactosidase