PMID: 9437492Jan 1, 1997Paper

Gene therapy: panacea or placebo? I. Strategies and limitations of gene therapy

Archivum Immunologiae Et Therapiae Experimentalis
D C Górecki, K D MacDermot

Abstract

Progress in genetics and molecular medicine has led to characterization of many disorders at the level of specific genes. Techniques for reliable diagnosis of these disorders have been developed in parallel. Attempts are currently being made to develop DNA-based therapeutic procedures to correct genetic diseases. These procedures are known under a common name of gene therapy. The initial step in gene therapy is the delivery of the gene of interest into target cells. There are several conceptually different approaches to achieve this, e.g. targeting can be accomplished by using viral vectors (transduction) or DNA-mediated routes (transformation) either ex vivo or in vivo. The selection of vector systems and the choice of delivering genes either into isolated cells or directly in the organism depend on factors like: the nature of target cell or organ, the levels of expression required, stability and/or regulation of expression, safety, etc. Many viruses have been adapted for use as vectors for gene therapy, according to their specific properties. Viral genomes have been modified to remove their ability to replicate and to increase the cloning capacity. Non-viral gene delivery systems rely on cellular mechanisms to import DNA into...Continue Reading

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