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Use of Manipulated Stem Cells for Prenatal Therapy

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Prenatal Gene Therapy

Part of the book series: Methods in Molecular Biology ((MIMB,volume 891))

Abstract

Prenatal stem cell therapy has broad potential for therapeutic application. “Stem cells” of interest include multipotent adult-derived stem cells, cord blood, amniotic fluid, or fetal stem cells, and embryonic or induced pluripotent stem cells. Potential manipulations of stem cells prior to their administration may include harvest, processing, enrichment, expansion, and genetic transduction. A complete description of the methodology related to all of the above is well beyond the scope of this chapter. In the interest of practical application and proven efficacy, we limit our description to adult-derived hematopoietic stem cells (HSCs) and their application to in utero transplantation with or without HSC-targeted gene transfer.

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References

  1. Spangrude GJ, Heimfeld S, Weissman IL (1988) Purification and characterization of mouse hematopoietic stem cells. Science 241:58–62

    Article  PubMed  CAS  Google Scholar 

  2. Flake AW (2004) The conceptual application of systems theory to stem cell biology: a matter of context. Blood Cells Mol Dis 32:58–64

    Article  PubMed  CAS  Google Scholar 

  3. Badillo AT, Flake AW (2007) The regulatory role of the stromal microenvironment in fetal hematopoietic ontogeny. Stem Cell Rev 2:241–246

    Article  Google Scholar 

  4. Laje P, Zoltick PW, Flake AW (2010) SLAM-enriched hematopoietic stem cells maintain long-term repopulating capacity after lentiviral transduction using an abbreviated protocol. Gene Ther 17(3):412–418

    Article  PubMed  CAS  Google Scholar 

  5. Flake AW, Zanjani ED (1999) In utero hematopoietic stem cell transplantation: ontogenic opportunities and biologic barriers. Blood 94:2179–2191

    PubMed  CAS  Google Scholar 

  6. Roybal JL, Santore MT, Flake AW (2010) Stem cell and genetic therapies for the fetus. Semin Fetal Neonatal Med 15:46–51

    Article  PubMed  Google Scholar 

  7. Endo M, Henriques-Coelho T, Zoltick PW et al (2010) The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors. Gene Ther 17:61–71

    Article  PubMed  CAS  Google Scholar 

  8. Christensen JL, Wright DE, Wagers AJ, Weissman IL (2004) Circulation and chemotaxis of fetal hematopoietic stem cells. PLoS Biol 2:E75

    Article  PubMed  Google Scholar 

  9. Merianos DJ, Tiblad E, Santore MT et al (2009) Maternal alloantibodies induce a postnatal immune response that limits engraftment following in utero hematopoietic cell transplantation in mice. J Clin Invest 119:2590–2600

    PubMed  CAS  Google Scholar 

  10. RAC (2000) Prenatal gene tranfer: scientific, medical, and ethical issues: a report of the Recombinant DNA Advisory Committee. Hum Gene Ther 11:1211–1229

    Article  Google Scholar 

  11. Themis M, Waddington SN, Schmidt M et al (2005) Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol Ther 12:763–771

    Article  PubMed  CAS  Google Scholar 

  12. Goodell MA, Brose K, Paradis G et al (1996) Isolation and functional properties of murine hematopoietic stem cells that are replicating in vivo. J Exp Med 183:1797–1806

    Article  PubMed  CAS  Google Scholar 

  13. Goodell MA, Rosenzweig M, Kim H et al (1997) Dye efflux studies suggest that hematopoietic stem cells expressing low or undetectable levels of CD34 antigen exist in multiple species. Nat Med 3:1337–1345

    Article  PubMed  CAS  Google Scholar 

  14. Kiel MJ, Yilmaz OH, Iwashita T et al (2005) SLAM family receptors distinguish hematopoietic stem and progenitor cells and reveal endothelial niches for stem cells. Cell 121:1109–1121

    Article  PubMed  CAS  Google Scholar 

  15. Yilmaz OH, Kiel MJ, Morrison SJ (2006) SLAM family markers are conserved among hematopoietic stem cells from old and reconstituted mice and markedly increase their purity. Blood 107:924–930

    Article  PubMed  CAS  Google Scholar 

  16. Randall TD, Weissman IL (1997) Phenotypic and functional changes induced at the clonal level in hematopoietic stem cells after 5-fluorouracil treatment. Blood 89:3596–3606

    PubMed  CAS  Google Scholar 

  17. Colvin GA, Lambert JF, Abedi M et al (2004) Murine marrow cellularity and the concept of stem cell competition: geographic and quantitative determinants in stem cell biology. Leukemia 18:575–583

    Article  PubMed  CAS  Google Scholar 

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Author information

Authors and Affiliations

  1. Department of Surgery, Children’s Center for Fetal Research and Center for Fetal Diagnosis and Treatment, Children’s Hospital of Philadelphia, Philadelphia, PA, USA

    Jessica L. Roybal, Pablo Laje & Jesse D. Vrecenak

  2. Department of Surgery, Children’s Hospital of Philadelphia, Philadelphia, PA, USA

    Alan W. Flake MD

Authors
  1. Jessica L. Roybal
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  2. Pablo Laje
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  3. Jesse D. Vrecenak
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  4. Alan W. Flake MD
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Corresponding author

Correspondence to Alan W. Flake MD .

Editor information

Editors and Affiliations

  1. National Heart & Lung Inst., Molecular & Cellular Medicine Sec., Imperial College London, Imperial College Road, London, SW7 2AZ, United Kingdom

    Charles Coutelle

  2. Reader in Gene Transfer Technology, Gene Transfer Technology Group, University College London, Chenies Mews 86-96, London, WC1E 6HX, United Kingdom

    Simon N. Waddington

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© 2012 Springer Science+Business Media, LLC.

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Roybal, J.L., Laje, P., Vrecenak, J.D., Flake, A.W. (2012). Use of Manipulated Stem Cells for Prenatal Therapy. In: Coutelle, C., Waddington, S. (eds) Prenatal Gene Therapy. Methods in Molecular Biology, vol 891. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-61779-873-3_8

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  • DOI: https://doi.org/10.1007/978-1-61779-873-3_8

  • Published:

  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-61779-872-6

  • Online ISBN: 978-1-61779-873-3

  • eBook Packages: Springer Protocols

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