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Related Experiment Videos

Long term physiologic modification using rAAV in utero gene-therapy.

Deiadra J Garrett1, J Craig Cohen, Janet E Larson

  • 1Departments of Medicine, Biochemistry, and Genetics, Louisiana State University Health Sciences Center, New Orleans, LA 70112, USA. ccohen@lsuhsc.edu

Genetic Vaccines and Therapy
|May 21, 2004
PubMed
Summary

In utero gene therapy using recombinant adeno-associated virus (rAAV) successfully modified rat physiology long-term. This approach shows potential for treating metabolic disorders through gene replacement therapy.

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Area of Science:

  • Regenerative Medicine
  • Gene Therapy
  • Developmental Biology

Background:

  • In utero gene transfer via amniotic fluid using recombinant adeno-associated viruses (rAAV) is highly efficient.
  • Previous studies demonstrated sustained gene expression and systemic protein release from transgenes delivered in utero.
  • These findings support the potential for long-term physiological modification through in utero gene therapy.

Purpose of the Study:

  • To test the hypothesis that in utero rAAV gene therapy can induce long-term physiological modifications.
  • To evaluate the efficacy of rAAV-mediated gene transfer for sustained therapeutic effects.
  • To explore the potential of this method for treating genetic disorders.

Main Methods:

  • A polycistronic rAAV vector expressing ciliary neurotrophic factor (CNTF) and green fluorescent protein (GFP) was used.

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  • Rat fetuses were treated in utero with the rAAV vector.
  • CNTF's known physiological effects (weight loss, decreased water intake) and GFP expression were monitored.

    • Main Results:

    • In utero gene transfer with rAAV resulted in sustained gene expression in rats for over one year.
    • CNTF-specific physiological effects, including decreased weight and water intake, were observed.
    • GFP expression confirmed successful gene transfer and expression in treated animals at one year of age.

    Conclusions:

    • In utero rAAV gene therapy targeting multipotential stem cells leads to long-term systemic physiological modification.
    • The transgene product mediated sustained physiological changes in treated animals.
    • In utero rAAV gene therapy holds promise for gene replacement therapy in metabolic disorders.