Use of manipulated stem cells for prenatal therapy

Methods in Molecular Biology

Volumn 891, Issue , 2012, Pages 169-181

  Roybal, Jessica L ^a   Laje, Pablo ^a   Vrecenak, Jesse D ^a   Flake, Alan W ^a  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

Gene transfer; Hematopoietic stem cell; In utero transplantation; Lentiviral vector

Indexed keywords

CD150 ANTIGEN; CELL SURFACE MARKER; LENTIVIRUS VECTOR; STEM CELL ANTIGEN 1; STEM CELL FACTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BONE MARROW; CELL ISOLATION; CELL SELECTION; EX VIVO GENE TRANSFER; EX VIVO STUDY; FLOW CYTOMETRY; HEMATOPOIETIC STEM CELL; IN UTERO STEM CELL TRANSPLANTATION; MONONUCLEAR CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; YOLK SAC;

EID: 84934434992     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-61779-873-3_8     Document Type: Article

References (17)

1. Spangrude GJ, Heimfeld S, Weissman IL (1988) Puri fi cation and characterization of mouse hematopoietic stem cells. Science 241:58-62
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
3. Badillo AT, Flake AW (2007) The regulatory role of the stromal microenvironment in fetal hematopoietic ontogeny. Stem Cell Rev 2: 241-246
4. Laje P, Zoltick PW, Flake AW (2010) SLAMenriched hematopoietic stem cells maintain long-term repopulating capacity after lentiviral transduction using an abbreviated protocol. Gene Ther 17(3):412-418
5. Flake AW, Zanjani ED (1999) In utero hematopoietic stem cell transplantation: ontogenic opportunities and biologic barriers. Blood 94:2179-2191
6. Roybal JL, Santore MT, Flake AW (2010) Stem cell and genetic therapies for the fetus. Semin Fetal Neonatal Med 15:46-51
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
8. Christensen JL, Wright DE, Wagers AJ, Weissman IL (2004) Circulation and chemotaxis of fetal hematopoietic stem cells. PLoS Biol 2:E75
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
10. RAC (2000) Prenatal gene tranfer: scienti fi c, medical, and ethical issues: a report of the Recombinant DNA Advisory Committee. Hum Gene Ther 11:1211-1229
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
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
13. Goodell MA, Rosenzweig M, Kim H et al (1997) Dye ef fl ux studies suggest that hematopoietic stem cells expressing low or undetectable levels of CD34 antigen exist in multiple species. Nat Med 3:1337-1345
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
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
16. Randall TD, Weissman IL (1997) Phenotypic and functional changes induced at the clonal level in hematopoietic stem cells after 5-fl uorouracil treatment. Blood 89:3596-3606
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