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Related Experiment Videos

Microcapsules as bio-organs for somatic gene therapy

P L Chang1

  • 1Department of Pediatrics, McMaster University, Hamilton, Ontario, Canada. changp@fhs.mcmaster.ca

Annals of the New York Academy of Sciences
|June 9, 1998
PubMed
Summary

This study demonstrates a novel gene therapy using encapsulated, genetically engineered cells to deliver therapeutic proteins. This approach shows promise for treating genetic diseases by providing a potentially cost-effective, non-autologous treatment option.

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Area of Science:

  • Biotechnology
  • Regenerative Medicine
  • Genetic Engineering

Background:

  • Current gene therapy often uses autologous cells, limiting accessibility and increasing costs.
  • Non-autologous, universal cell lines offer a potential alternative for widespread therapeutic applications.
  • Immuno-isolation devices can protect engineered cells, enabling shared use across patients.

Purpose of the Study:

  • To evaluate the feasibility and efficacy of using encapsulated, genetically modified cells for delivering therapeutic gene products.
  • To demonstrate the potential of a non-autologous gene therapy approach for treating genetic disorders.
  • To explore the use of bioartificial devices for sustained delivery of recombinant proteins.

Main Methods:

  • Genetically modified cells (fibroblasts, myoblasts) were engineered to express therapeutic proteins like human growth hormone and factor IX.

Related Experiment Videos

  • Cells were encapsulated using alginate-poly-L-lysine-alginate or hydroxyethyl methacrylate-methyl methacrylate for immuno-isolation.
  • Encapsulated cells were implanted intraperitoneally in murine models of human genetic diseases (Snell dwarf mice, Gus/Gus mice).

    • Main Results:

    • Successful expression and systemic delivery of recombinant human growth hormone and factor IX from encapsulated cells in vivo.
    • Implantation of encapsulated, growth hormone-secreting cells in Snell dwarf mice led to significant increases in body weight and organ size.
    • Encapsulated, beta-glucuronidase-secreting cells in Gus/Gus mice normalized plasma enzyme levels and corrected histopathology.

    Conclusions:

    • Delivery of recombinant gene products via encapsulated, genetically engineered cells is a feasible and effective strategy for gene therapy.
    • This bioartificial device approach offers a promising, potentially cost-effective alternative to traditional gene therapy methods.
    • The study validates the therapeutic potential of immuno-isolated, engineered cells for treating various genetic diseases.