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Gene Therapy: Vector Design and Current Challenges

  • Elizabeth G. Nabel
Part of the Developments in Cardiovascular Medicine book series (DICM, volume 193)

Abstract

Gene transfer is an approach to the treatment of human diseases based on the delivery and expression of genetic material into somatic cells. Gene delivery can be achieved in vivo through direct introduction into cells or ex vivo through manipulation of cells in culture. In this sense, gene therapy is a new form of drug delivery, in which the drug is recombinant protein synthesized by transduced cells. Gene therapy can be viewed as an extension of conventional medical therapies in which the genetic material is the therapeutic agent. Gene therapy is being applied to many human diseases, and it offers the potential to cure diseases, either as a single treatment modality or as an adjuvant to existing treatments. However, gene therapy is still in its infancy, and many of these promises have not yet been fulfilled.

Keywords

Cystic Fibrosis Gene Therapy Gene Delivery Peripheral Artery Disease Nonviral Vector 
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

© Kluwer Academic Publishers 1997

Authors and Affiliations

  • Elizabeth G. Nabel

There are no affiliations available

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