Gene Therapy Chapter uri icon


MeSH Major

  • Continuous Positive Airway Pressure
  • Pneumothorax


  • Gene transfer is a therapeutic strategy which uses genetic information, usually in the form of DNA, to modify the phenotype of cells. Gene therapy strategies can be useful for tissue engineering by modifying cells directly or providing a favorable growth environment for the engineered tissue. To accomplish this, cells are modified genetically ex vivo or in vivo using gene transfer vectors that mediate the transfer of therapeutic DNA into the nucleus where it is transcribed in parallel with genomic DNA. A variety of non-viral and viral gene therapy vectors have been developed, including plasmids, plasmids combined with liposomes, adenovirus, adeno-associated virus (AAV), retrovirus and lentivirus. The two current vector systems which are most promising for the treatment of genetic diseases are AAV for in vivo gene transfer to postmitotic cells, and lentiviral vectors for ex vivo gene transfer to stem cells and hematopoietic cells. A number of strategies have evolved to enhance the targeting of gene transfer vectors by genetic or chemical modification of the surface of the vector. Gene expression directed by the transferred gene can be regulated by including inducible promoters, tissue-specific promoters and trans-splicing. There is significant potential for combining gene therapy with stem cell strategies to aid in controlling cell growth and circumventing immune rejection, and the transfer of genes for reprogramming factors is one strategy for generating induced pluripotent stem cells. There are still challenges for using gene transfer for tissue engineering, but the technology of gene transfer is sufficiently advanced to be an important part of tissue-engineering strategies. © 2014 Elsevier Inc. All rights reserved.

publication date

  • November 2013



  • Book Chapter


Digital Object Identifier (DOI)

  • 10.1016/B978-0-12-398358-9.00034-3

Additional Document Info

start page

  • 657

end page

  • 686