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Pharmaceutical approach to somatic gene therapy

F D Ledley1

  • 1GENEMEDICINE, INC., Woodlands, Texas 77381, USA.

Pharmaceutical Research
|November 1, 1996
PubMed
Summary

Somatic gene therapy views genes as chemical entities. Advanced pharmaceutical drug delivery systems are emerging to overcome DNA

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Area of Science:

  • Pharmaceutical sciences
  • Molecular biology
  • Biotechnology

Background:

  • Somatic gene therapy treats diseases by modifying genes.
  • Genes are large, negatively charged molecules (>1x10^6 Daltons, >100 nm diameter).
  • These properties hinder diffusion through biological barriers like cell and nuclear membranes.

Purpose of the Study:

  • To review current gene therapy approaches.
  • To explore the emerging pharmaceutical perspective on gene therapy.
  • To highlight advanced drug delivery methods for gene therapy.

Main Methods:

  • Review of existing gene therapy administration methods (cells, viruses, plasmid DNA).
  • Analysis of DNA's physical, chemical, and colloidal properties.
  • Examination of advanced drug delivery systems and particulate systems.
  • Discussion of controlling gene expression with recombined genetic elements.

Main Results:

  • Current gene therapy relies on cells, viruses, and plasmid DNA.
  • An emerging pharmaceutical approach focuses on DNA's inherent properties.
  • Advanced drug delivery systems are crucial for overcoming biological barriers.
  • Understanding cellular uptake and nuclear trafficking pathways is key.

Conclusions:

  • The pharmaceutical approach offers new strategies for somatic gene therapy.
  • Advanced drug delivery is essential for effective gene therapy.
  • Integrating pharmaceutical principles enhances gene therapy development.

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