Genetic modification of stem cells for cardiac, diabetic, and hemophilia transplantation therapies

M. Ian Phillips, Yaoliang Tang

Research output: Chapter in Book/Report/Conference proceedingChapter

7 Scopus citations

Abstract

Gene modification of stem cells prior to their transplantation enhances their survival and increases their function in cell therapy. Like the famous Trojan horse, the gene-modified cell has to gain entrance into the host's walls and survive to deliver its transgene products. Using cellular, molecular, and gene manipulation techniques, the transplanted cell can be protected in a hostile environment from immune rejection, inflammation, hypoxia, and apoptosis. Genetic engineering to modify cells involves construction of functional gene sequences and their insertion into stem cells. The modifications can be simple reporter genes or complex cassettes with gene switches, cell-specific promoters, and multiple transgenes. We discuss methods to deliver and construct gene cassettes with viral and nonviral delivery, siRNA, and conditional Cre/Lox P. We review the current uses of gene-modified stem cells in cardiovascular disease, diabetes, and hemophilia.

Original languageEnglish (US)
Title of host publicationProgress in Molecular Biology and Translational Science
PublisherElsevier B.V.
Pages285-304
Number of pages20
DOIs
StatePublished - 2012
Externally publishedYes

Publication series

NameProgress in Molecular Biology and Translational Science
Volume111
ISSN (Print)1877-1173

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Keywords

  • Cre/LoxP
  • Diabetes
  • Heart failure
  • Hemophilia
  • MicroRNA
  • Vigilant vector
  • siRNA

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology

Cite this

Phillips, M. I., & Tang, Y. (2012). Genetic modification of stem cells for cardiac, diabetic, and hemophilia transplantation therapies. In Progress in Molecular Biology and Translational Science (pp. 285-304). (Progress in Molecular Biology and Translational Science; Vol. 111). Elsevier B.V.. https://doi.org/10.1016/B978-0-12-398459-3.00013-7