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Related Experiment Videos

Gene therapy for newborns

D B Kohn1, R Parkman

  • 1Childrens Hospital Los Angeles, and Department of Pediatrics, University of Southern California School of Medicine, 90027, USA.

FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology
|July 1, 1997
PubMed
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Gene therapy in newborns shows promise for treating genetic diseases by modifying umbilical cord blood stem cells. This approach successfully corrected a congenital immune deficiency, offering potential for other inherited and infectious disorders.

Area of Science:

  • Neonatal medicine
  • Gene therapy
  • Stem cell biology

Background:

  • Gene therapy offers potential advantages in newborns due to minimal disease impact and growth potential.
  • Umbilical cord blood provides a unique target for hematopoietic stem cell modification.
  • Neonatal gene therapy can address congenital immune deficiencies and other disorders.

Purpose of the Study:

  • To evaluate the efficacy of gene therapy in newborns.
  • To assess the potential of using umbilical cord blood for gene modification.
  • To explore gene therapy applications for congenital immune deficiencies and infectious diseases.

Main Methods:

  • Gene insertion of a normal adenosine deaminase gene into umbilical cord blood cells.
  • Application in three neonates diagnosed with congenital immune deficiency.

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  • Long-term monitoring of stem cell engraftment and leukocyte production.

    • Main Results:

    • Successful transduction and sustained engraftment of modified hematopoietic stem cells.
    • Demonstrated contribution to leukocyte production for over three years post-treatment.
    • Established proof-of-concept for neonatal gene therapy using cord blood.

    Conclusions:

    • Gene therapy in newborns using umbilical cord blood is feasible and effective for congenital immune deficiencies.
    • This approach holds potential for treating other neonatal genetic and infectious diseases, including HIV-1.
    • Prenatal diagnosis and regulatory compliance are crucial for expanding neonatal gene therapy applications.