The pharmacology of gene therapy

Minchin, Rodney F., Orr, Rebecca J., Cronin, Andrew S. and Puls, Rebecca L. (1999) The pharmacology of gene therapy. Croatian Medical Journal, 40 3: 381-391.

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Author Minchin, Rodney F.
Orr, Rebecca J.
Cronin, Andrew S.
Puls, Rebecca L.
Title The pharmacology of gene therapy
Journal name Croatian Medical Journal   Check publisher's open access policy
ISSN 0353-9504
Publication date 1999-09-01
Sub-type Critical review of research, literature review, critical commentary
Open Access Status File (Publisher version)
Volume 40
Issue 3
Start page 381
End page 391
Total pages 11
Place of publication Zagreb, Croatia
Publisher Medicinska Naklada
Language eng
Formatted abstract
The objective for human gene therapy is to express exogenous DNA at a site in vivo for long enough, and at sufficient levels to produce a therapeutic response. The obstacles to this objective are numerous and include the formulation or packaging of the DNA, in vivo delivery, penetration of biological barriers, DNA elimination within the cell and from the tissue compartments of the whole body, control of product expression and overt toxicity. The current challenge is to resolve each of these obstacles to produce a practical and efficient gene therapy. In doing so, it is vital to understand the disposition of DNA vectors in vivo, and to know how conventional medicines may be used to modulate this disposition and to enhance the therapeutic effect of these vectors. Many of the general concepts of human gene therapy have been reviewed extensively in the literature. This review discusses some of the pharmacological aspects of gene delivery and the fate of vectors in vivo, and then highlights how drugs are being used to modulate gene therapy.
Keyword Ganciclovir
Gene targeting
Genetic engineering
Gene therapy
Gene transfer
Regulation of gene expression
Somatic gene therapy
Vectors, genetic
Medicine, General and Internal
Q-Index Code C1
Q-Index Status Provisional Code
Institutional Status Non-UQ

Document type: Journal Article
Sub-type: Critical review of research, literature review, critical commentary
Collection: School of Biomedical Sciences Publications
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Citation counts: TR Web of Science Citation Count  Cited 5 times in Thomson Reuters Web of Science Article | Citations
Scopus Citation Count Cited 5 times in Scopus Article | Citations
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Created: Mon, 13 Aug 2007, 21:21:54 EST