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New Tools in Regenerative Medicine: Gene Therapy

  • Miguel Muñoz Ruiz
  • José R. RegueiroEmail author
Part of the Advances in Experimental Medicine and Biology book series (AEMB, volume 741)

Abstract

Gene therapy aims to transfer genetic material into cells to provide them with new functions. A gene transfer agent has to be safe, capable of expressing the desired gene for a sustained period of time in a sufficiently large population of cells to produce a biological effect. Identifying a gene transfer tool that meets all of these criteria has proven to be a difficult objective. Viral and nonviral vectors, in vivo, ex vivo and in situ strategies co-exist at present, although ex vivo lenti- or retroviral vectors are presently the most popular.

Natural stem cells (from embryonic, hematopoietic, mesenchymal, or adult tissues) or induced progenitor stem (iPS) cells can be modified by gene therapy for use in regenerative medicine. Among them, hematopoietic stem cells have shown clear clinical benefit, but iPS cells hold humongous potential with no ethical concerns.

Keywords

Stem Cell Gene Therapy Mesenchymal Stem Cell Regenerative Medicine Chronic Granulomatous Disease 
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

© Landes Bioscience and Springer Science+Business Media 2012

Authors and Affiliations

  1. 1.Department of ImmunologyUniversidad Complutense de MadridMadridSpain

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