Genetically engineered mesenchymal stem cells for cell and gene therapy

Yunjoon Jung, Jan Nolta

Research output: Chapter in Book/Report/Conference proceedingChapter


Stem cells have enormous potential for regenerative medicine to treat fatal diseases and injuries that cannot otherwise be healed. In particular, adult stem cell-based therapies have been studied for several decades. Mesenchymal stem cells/marrow stromal cells (MSCs) have shown safety and therapeutic efficacy in preclinical models of various diseases such as cardiovascular disease, cancer, bone defects, renal failure, and neurodegenerative disorders. In spite of the great potential, several factors including low survival rate, low efficiency of MSC homing to injured sites, and poor levels of engraftment and retention have been major technical challenges to be overcome before MSC-based therapy can be applied to clinical applications in a consistently therapeutic manner. Genetically modified MSCs can be one option to overcome some of these problems and to deliver therapeutic agents. MSCs are powerful delivery vehicles and potent protein synthesis factories, and therefore the use of gene-modified MSCs to provide growth factors and other signals to improve the repair of damaged or diseased tissues holds much promise. Here we review the basic biology of human MSCs and the current status of preclinical and clinical trials using genetically engineered MSCs.

Original languageEnglish (US)
Title of host publicationMesenchymal Stem Cell Therapy
PublisherHumana Press Inc.
Number of pages34
ISBN (Electronic)9781627032001
ISBN (Print)9781627031998
StatePublished - Jan 1 2013


  • Bone disease
  • Cancer
  • Cellular therapy
  • Clinical trials
  • Gene therapy
  • Growth factor production
  • Mesenchymal stem cells
  • Neurodegenerative disorders
  • Preclinical models
  • Regenerative medicine
  • Renal disease
  • Revascularization

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)


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