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Vectors--shuttle vehicles for gene therapy

J M Wilson1

  • 1Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia 19104-4268, USA.

Clinical and Experimental Immunology
|January 1, 1997
PubMed
Summary
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Gene therapy uses gene delivery vehicles, or vectors, to treat diseases. Viral vectors like adenoviruses show promise for in vivo gene therapy, enabling gene transfer into non-dividing cells for various conditions.

Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Gene therapy aims to treat disorders by manipulating gene expression.
  • Early gene therapy focused on ex vivo manipulation of autologous cells.
  • In vivo gene therapy involves direct gene delivery to cells within the body.

Purpose of the Study:

  • To review the advancements in gene delivery vehicles for gene therapy.
  • To compare ex vivo and in vivo gene therapy approaches.
  • To highlight the potential of viral vectors, specifically adenoviruses, for in vivo applications.

Main Methods:

  • Utilized recombinant viral vectors, including retroviral and adenoviral types.
  • Compared the efficacy of ex vivo gene therapy using retroviral vectors with dividing cells.

Related Experiment Videos

  • Evaluated in vivo gene therapy using adenoviral vectors for transducing non-dividing cells.

    • Main Results:

    • Retroviral vectors are effective for ex vivo gene therapy in dividing cells but limited for in vivo use.
    • Adenoviral vectors are suitable for in vivo gene therapy due to their ability to transduce non-dividing cells.
    • Adenoviruses can be produced in large quantities and highly purified, facilitating in vivo gene delivery.

    Conclusions:

    • Adenoviral vectors represent a significant advancement for in vivo gene therapy.
    • In vivo gene therapy holds potential for treating diseases affecting various organs like the lung, liver, and central nervous system.
    • Clinical trials using adenoviruses for in vivo gene delivery, such as in cystic fibrosis treatment, are underway.