Targeted vectors for gene therapy

Research output: Contribution to journalArticle

148 Citations (Scopus)

Abstract

Successful gene therapy requires not only the identification of an appropriate therapeutic gene for treatment of the disease, but also a delivery system by which that gene can be delivered to the desired cell type both efficiently and accurately. Reductions in accuracy will inevitably also reduce efficiency since fewer particles will be available for delivery to the correct cells if many are sequestered into nontarget cells. In addition, the therapy will have net benefit to the patient only if gene delivery is sufficiently restricted such that normal calls are left unaffected by any detrimental affects of bystander cell transduction. Here we review how currently available delivery systems, both plasmid and viral, can be manipulated to improve their targeting to specific cell types. Currently, targeting is achieved by engineering of the surface components of viruses and liposomes to achieve discrimination at the level of target cell recognition and/or by incorporating transcriptional elements into plasmid or vital genomes such that the therapeutic gene is expressed only in certain target cell types. In addition, we discuss emerging vectors and suggest how gene therapy delivery systems of the future will be composites of the best features of diverse vectors already in use.

Original languageEnglish (US)
Pages (from-to)190-199
Number of pages10
JournalFASEB Journal
Volume9
Issue number2
StatePublished - 1995
Externally publishedYes

Fingerprint

Gene therapy
gene therapy
Genetic Therapy
Genes
Gene Transfer Techniques
cells
Plasmids
therapeutics
plasmids
Viruses
Liposomes
Viral Structures
genes
Therapeutics
gene transfer
engineering
Composite materials
Genome
viruses
genome

ASJC Scopus subject areas

  • Agricultural and Biological Sciences (miscellaneous)
  • Biochemistry, Genetics and Molecular Biology(all)
  • Biochemistry
  • Cell Biology

Cite this

Targeted vectors for gene therapy. / Miller, N.; Vile, Richard Geoffrey.

In: FASEB Journal, Vol. 9, No. 2, 1995, p. 190-199.

Research output: Contribution to journalArticle

Miller, N & Vile, RG 1995, 'Targeted vectors for gene therapy', FASEB Journal, vol. 9, no. 2, pp. 190-199.
Miller, N. ; Vile, Richard Geoffrey. / Targeted vectors for gene therapy. In: FASEB Journal. 1995 ; Vol. 9, No. 2. pp. 190-199.
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