Gene Therapy

Stefan Worgall, Ronald G. Crystal

Research output: Chapter in Book/Report/Conference proceedingChapter

1 Citation (Scopus)


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.

Original languageEnglish
Title of host publicationPrinciples of Tissue Engineering
Subtitle of host publicationFourth Edition
PublisherElsevier Inc.
Number of pages30
ISBN (Print)9780123983589
Publication statusPublished - 1 Nov 2013



  • AAV
  • Adeno-associated virus
  • Adenovirus
  • Gene therapy
  • Gene transfer
  • Genomic DNA
  • IPS
  • Lentivirus
  • Liposomes
  • Non-viral
  • Parallel
  • Phenotype
  • Plasmids
  • Pluripotent stem cells
  • Retrovirus
  • Therapeutic DNA
  • Viral

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

Cite this

Worgall, S., & Crystal, R. G. (2013). Gene Therapy. In Principles of Tissue Engineering: Fourth Edition (pp. 657-686). Elsevier Inc..