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Dual-regulated gene expression in mammalian cells, a novel approach to gene therapy

Dual-regulated gene expression in mammalian cells, a novel approach to gene therapy Gene Therapy and Regulation , Vol. 1, No. 3, pp. 233– 264 (2001) Ó VSP 2001. Dual-regulated gene expression in mammalian cells, a novel approach to gene therapy * MARTIN FUSSENEGGER † Institute of Biotechnology, Swiss Federal Institute of Technology, ETH Zurich, CH-8093 Zurich, Switzerland Received 13 March 2001 Abstract —A variety of heterologous mammalian gene regulation systems are currently available for use in human gene therapy and tissue engineering. While individual gene regulation concepts vary signiŽ cantly in key characteristics (regulation proŽ les, basal expression levels, human compatibility, immunogenicity, pharmacokinetics and bioavailability of the inducing agent), most of them are sufŽ ciently elaborated to be considered for preclinical research and clinical gene therapy trials. As forefront basic research describes the cell-cycle, differentiation and apoptosis regulatory networks in mammalian cells as an increasingly complex highly interconnected globular regulon, it becomes apparent that many human diseases may originate from the slightest expression imbalances of key regulatory genes. We believe that successful gene therapy of such diseases will involve complex multi-(level) regulated multigene interventions based on a combination of several human-compatible heterologous gene regulation systems which enable optimal integration of therapeutic interventions into the cellular regulon as well as well-balanced http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Gene Therapy and Regulation Brill

Dual-regulated gene expression in mammalian cells, a novel approach to gene therapy

Fussenegger, Martin
Gene Therapy and Regulation , Volume 1 (3): 233 – Jan 1, 2001
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Publisher
Brill
Copyright
© 2000 Koninklijke Brill NV, Leiden, The Netherlands
ISSN
1388-9532
eISSN
1568-5586
DOI
10.1163/156855800750367446
Publisher site
See Article on Publisher Site

Abstract

Gene Therapy and Regulation , Vol. 1, No. 3, pp. 233– 264 (2001) Ó VSP 2001. Dual-regulated gene expression in mammalian cells, a novel approach to gene therapy * MARTIN FUSSENEGGER † Institute of Biotechnology, Swiss Federal Institute of Technology, ETH Zurich, CH-8093 Zurich, Switzerland Received 13 March 2001 Abstract —A variety of heterologous mammalian gene regulation systems are currently available for use in human gene therapy and tissue engineering. While individual gene regulation concepts vary signiŽ cantly in key characteristics (regulation proŽ les, basal expression levels, human compatibility, immunogenicity, pharmacokinetics and bioavailability of the inducing agent), most of them are sufŽ ciently elaborated to be considered for preclinical research and clinical gene therapy trials. As forefront basic research describes the cell-cycle, differentiation and apoptosis regulatory networks in mammalian cells as an increasingly complex highly interconnected globular regulon, it becomes apparent that many human diseases may originate from the slightest expression imbalances of key regulatory genes. We believe that successful gene therapy of such diseases will involve complex multi-(level) regulated multigene interventions based on a combination of several human-compatible heterologous gene regulation systems which enable optimal integration of therapeutic interventions into the cellular regulon as well as well-balanced

Journal

Gene Therapy and Regulation – Brill

Published: Jan 1, 2001

Keywords: STREPTOGRAMIN-RESPONSIVE EXPRESSION TECHNOLOLOGY; HETEROLOGOUS GENE REGULATION; TISSUE ENGINEERING; GENE THERAPY

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