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Open Access Green as soon as Postprint is submitted to ZB.
Episomal vectors for gene therapy.
Curr. Gene Ther. 8, 147-161 (2008)
The increasing knowledge of the molecular and genetic background of many different human diseases has led to the vision that genetic engineering might be used one day for their phenotypic correction. The main goal of gene therapy is to treat loss-of-function genetic disorders by delivering correcting therapeutic DNA sequences into the nucleus of a cell, allowing its long-term expression at physiologically relevant levels. Manifold different vector systems for the therapeutic gene delivery have been described over the recent years. They all have their individual advantages but also their individual limitations and must be judged on a careful risk/benefit analysis. Integrating vector systems can deliver genetic material to a target cell with high efficiency enabling long-term expression of an encoded transgene. The main disadvantage of integrating vector systems, however, is their potential risk of causing insertional mutagenesis. Episomal vector systems have the potential to avoid these undesired side effects, since they behave as separate extrachromosomal elements in the nucleus of a target cell. Within this article we present a comprehensive survey of currently available episomal vector systems for the genetic modification of mammalian cells. We will discuss their advantages and disadvantages and their applications in the context of basic research, biotechnology and gene therapy.
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Publication type
Article: Journal article
Document type
Scientific Article
Keywords
gene therapy; episomal vectors; vector administration; vector replication; nuclear retention; virus-like particles; naked DNA delivery; artificial chromosomes
Language
english
Publication Year
2008
HGF-reported in Year
0
ISSN (print) / ISBN
1566-5232
e-ISSN
1875-5631
Journal
Current Gene Therapy
Quellenangaben
Volume: 8,
Issue: 3,
Pages: 147-161
Publisher
Bentham Science Publishers
Reviewing status
Peer reviewed
Institute(s)
Research Unit Gene Vector (AGV)
POF-Topic(s)
30203 - Molecular Targets and Therapies
Research field(s)
Immune Response and Infection
PSP Element(s)
G-501500-001
WOS ID
000257206300001
Erfassungsdatum
2008-12-15