Abstract

The Stem Cell Core will provide two main functions for the Gene Therapy Program Projects. Marrow, peripheral blood and cord blood pluripotent hematopoietic stem cells (PHSCs) will be isolated by monoclonal antibody staining and fluorescence activated cell sorting. In addition, this core will perform in vitro and in vivo assays to detect pluripotent hematopoietic stem cells. These assays will include in vitro long term bone marrow cultures which detect the long-term culture-initiating cell which is the best qualitative in vitro assay for PHSCs. In addition, both the SCID-hu bone and thymus will serve as in vivo assays of PHSC function. Analysis of BFU-E cloned from the long-term marrow cultures and red blood cells isolated from IL-3 and erythropoietin primed SCID-hu bone assays will allow us to determine if new genes are being successfully transferred into erythroid cells which originate from transduced PHSCs. This core will also provide advice in training investigators that require access to flow cytometry or as says of hematopoietic progenitor or stem cells.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
5P01HL053762-03
Application #
5214254
Study Section
Project Start
Project End
Budget Start
Budget End
Support Year
3
Fiscal Year
1996
Total Cost
Indirect Cost

  Publications

Mizunoe, Shota; Shuto, Tsuyoshi; Suzuki, Shingo et al. (2012) Synergism between interleukin (IL)-17 and Toll-like receptor 2 and 4 signals to induce IL-8 expression in cystic fibrosis airway epithelial cells. J Pharmacol Sci 118:512-520
Ye, Lin; Chang, Judy C; Lu, Ronghua et al. (2008) High oxygen environment during pregnancy rescues sickle cell anemia mice from prenatal death. Blood Cells Mol Dis 41:67-72
Han, Xiao-Dong; Lin, Chin; Chang, Judy et al. (2007) Fetal gene therapy of alpha-thalassemia in a mouse model. Proc Natl Acad Sci U S A 104:9007-11
Chang, Judy C; Ye, Lin; Kan, Yuet Wai (2006) Correction of the sickle cell mutation in embryonic stem cells. Proc Natl Acad Sci U S A 103:1036-40
Jimenez, D F; Lee, C I; O'Shea, C E et al. (2005) HIV-1-derived lentiviral vectors and fetal route of administration on transgene biodistribution and expression in rhesus monkeys. Gene Ther 12:821-30
Jimenez, Daniel F; Leapley, Alyssa C; Lee, Chang I et al. (2005) Fetal CD34+ cells in the maternal circulation and long-term microchimerism in rhesus monkeys (Macaca mulatta). Transplantation 79:142-6
Jimenez, Daniel F; Tarantal, Alice F (2003) Quantitative analysis of male fetal DNA in maternal serum of gravid rhesus monkeys (Macaca mulatta). Pediatr Res 53:18-23
Meiering, C D; Comstock, K E; Linial, M L (2000) Multiple integrations of human foamy virus in persistently infected human erythroleukemia cells. J Virol 74:1718-26
Lipshutz, G S; Sarkar, R; Flebbe-Rehwaldt, L et al. (1999) Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero. Proc Natl Acad Sci U S A 96:13324-9
Lipshutz, G S; Flebbe-Rehwaldt, L; Gaensler, K M (1999) Adenovirus-mediated gene transfer to the peritoneum and hepatic parenchyma of fetal mice in utero. Surgery 126:171-7

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