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Unlocking Mechanisms in Gene Therapy, Stress and Proteomics

  • Andrew D. Miller
Conference paper
Part of the NATO Science Series book series (NAII, volume 129)

Abstract

Gene therapy may be described as the use of genes as medicines to treat disease, or more precisely as the delivery of nucleic acids by means of a vector to patients for some therapeutic purpose. Gene therapy is a therapeutic modality with enormous promise, but one that has regrettably failed to deliver much of therapeutic significance to date in spite of substantial clinical trial interest throughout the world [1]. General inadequacy of the vector systems used to deliver therapeutic nucleic acids to desired sites of action is the primary reason for lack of clinical success. Researchers have been seduced by the apparent simplicity of gene therapy approaches to treatment driving for clinical applications before vector technologies have been adequately developed or understood. Predictably, there has been a dramatic loss of confidence in gene therapy research in recent times matched by a decline in scientific and public perceptions of gene therapy. In my view this is unhelpful, gene therapy retains all future promise but there now needs to be a period of patient, logical technical and scientific development of new vector systems prior to any major second round of clinical trial activity [1].

Keywords

Gene Therapy Cystic Fibrosis Transmembrane Conductance Regulator Cationic Liposome Nuclear Localisation Sequence Cystic Fibrosis Mouse 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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Copyright information

© Springer Science+Business Media Dordrecht 2003

Authors and Affiliations

  • Andrew D. Miller
    • 1
  1. 1.Department of ChemistryImperial College London Imperial College Genetic Therapies CentreSouth Kensington, LondonUK

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