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Genetically Engineered Mesenchymal Stem Cells for Cell and Gene Therapy

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Mesenchymal Stem Cell Therapy

Part of the book series: Stem Cell Biology and Regenerative Medicine ((STEMCELL))

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Abstract

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.

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Acknowledgement

We thank the California Institute for Regenerative Medicine (CIRM), National Institute of Health (NIH), and philanthropic donors for supporting our research. We apologize to our colleagues whose work could not be cited due to the space limitation.

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Authors and Affiliations

  1. Department of Biomedical Engineering, University of California, Davis, Sacramento, CA, 95817, USA

    Yunjoon Jung

  2. Stem Cell Program and Institute for Regenerative Cures, University of California, Davis, Sacramento, CA, 95817, USA

    Yunjoon Jung & Jan A. Nolta Ph.D. (Director)

  3. Stem Cell Program and Institute for Regenerative Cures Scientific Director, UC Davis GMP facility Editor, Stem Cells University of California, Davis, 2921 Stockton Blvd., Room 1300, Sacramento, CA, 95817, USA

    Jan A. Nolta Ph.D. (Director)

  4. Division of Hematology/Oncology, Department of Internal Medicine, University of California, Davis, Sacramento, CA, 95817, USA

    Jan A. Nolta Ph.D. (Director)

Authors
  1. Yunjoon Jung
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  2. Jan A. Nolta Ph.D.
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Correspondence to Jan A. Nolta Ph.D. .

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Editors and Affiliations

  1. Cellular Dynamics International, Inc., Science Drive 525, Madison, 53711, Wisconsin, USA

    Lucas G. Chase

  2. , Stem Cells and Regenerative Medicine, Life Technologies, Executive Way 7335, Frederick, 21704, Maryland, USA

    Mohan C. Vemuri

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Jung, Y., Nolta, J.A. (2013). Genetically Engineered Mesenchymal Stem Cells for Cell and Gene Therapy. In: Chase, L., Vemuri, M. (eds) Mesenchymal Stem Cell Therapy. Stem Cell Biology and Regenerative Medicine. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-62703-200-1_15

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