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Prospects for human gene therapy.

E M Karson1, W Polvino, W F Anderson

  • 1Molecular Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892.

The Journal of Reproductive Medicine
|June 1, 1992
PubMed
Summary
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Gene transfer into cultured cells and fetal cord blood shows potential for treating genetic diseases. Early prenatal gene therapy in sheep demonstrated long-term marker gene expression after birth.

Area of Science:

  • * Molecular Biology
  • * Gene Therapy
  • * Developmental Biology

Background:

  • * Retroviral vectors are utilized for gene transfer into cultured lymphocytes for patient reinfusion.
  • * Hematopoietic progenitor cells (HPCs) from human fetal cord blood can be transduced with marker genes and human protein sequences.
  • * Ex vivo gene transduction of fetal sheep cord blood cells followed by in utero transfusion is a model for prenatal gene therapy.

Observation:

  • * Transduced genes were expressed in vitro by circulating hematopoietic progenitor cells from human fetal cord blood.
  • * Fetal sheep transfused with ex vivo transduced cord blood cells expressed marker genes for up to two years post-birth.

Findings:

  • * Successful in vitro gene transduction of human fetal cord blood HPCs.

Related Experiment Videos

  • * In utero gene therapy in sheep resulted in sustained marker gene expression postnatally.

    • Implications:

    • * Potential for treating genetic diseases through prenatal gene therapy, either in utero or shortly after birth.
    • * Highlights the need for improved gene transfer efficiency and long-term expression for clinical application.
    • * Suggests cord blood stem cells as a viable target for prenatal genetic interventions.