Genetic Manipulation Via Gene Transfer

Paul C. Edwards, Michael J. Passineau

Research output: Chapter in Book/Report/Conference proceedingChapter


Gene transfer is a potentially powerful approach to stem cell biology and tissue engineering, by engaging and altering the software of cellular behavior. While drug or chemical-based therapeutics can certainly modify cellular behavior, no method matches the potential of gene transfer for precise manipulation of cellular programming.The target tissues for craniofacial gene transfer therapy discussed in this chapter bear great similarity to those tissues where the greatest progress in clinical gene therapy has already been demonstrated (e.g., eye, muscle, skin, and bone), underscoring the potential power of gene transfer in dental and craniofacial therapeutics.Several proof-of-concept ex vivo and in vivo gene transfer strategies have been employed in pre-clinical studies, with numerous other prospective in vivo gene transfer approaches for the management of pathologic processes involving the oral and maxillofacial region at earlier stages of development. However, recognizing the many necessary safety and regulatory issues involved in the development of any gene transfer study, it is not surprising that the few successful stage 3 gene transfer trials to date have largely focused on the treatment of rare single gene Mendelian-inherited diseases. Nevertheless, as illustrated in this chapter, the potential conditions and approaches to gene therapy for conditions involving the head and neck complex are wide-ranging.

Original languageEnglish (US)
Title of host publicationStem Cell Biology and Tissue Engineering in Dental Sciences
PublisherElsevier Inc.
Number of pages10
ISBN (Electronic)9780123977786
ISBN (Print)9780123971579
StatePublished - Jan 1 2015


  • Clinical trials
  • Gene transfer
  • Genetic manipulation
  • Human gene therapy

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

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