Genetic modification of cells for transplantation

Adv Drug Deliv Rev. 2008 Jan 14;60(2):146-59. doi: 10.1016/j.addr.2007.08.039. Epub 2007 Oct 18.

Abstract

Progress in gene therapy has produced promising results that translate experimental research into clinical treatment. Gene modification has been extensively employed in cell transplantation. The main barrier is an effective gene delivery system. Several viral vectors were utilized in end-stage differentiated cells. Recently, successful applications were described with adenovirus-associated vectors. As an alternative, embryonic stem cell- and stem cell-like systems were established for generation of tissue-specified gene-modified cells. Owing to the feasibility for genetic manipulations and the self-renewing potency of these cells they can be used in a way enabling large-scale in vitro production. This approach offers the establishment of in vitro cell culture systems that will deliver sufficient amounts of highly purified, immunoautologous cells suitable for application in regenerative medicine. In this review, the current technology of gene delivery systems to cells is recapitulated and the latest developments for cell transplantation are discussed.

Publication types

  • Review

MeSH terms

  • Animals
  • Cell Culture Techniques / methods
  • Cell Differentiation / genetics
  • Cell Transplantation / methods*
  • Embryonic Stem Cells / cytology
  • Embryonic Stem Cells / transplantation
  • Gene Transfer Techniques*
  • Genetic Therapy / methods
  • Genetic Vectors / genetics
  • Humans