Abstract

The present proposal is for continued funding for a Specialized Center of Research in Hematopoietic Stem Cell Biology at the Children's Hospital Long Angeles (CHLA) and the University of California Los Angeles (UCLA). The programmatic focus of the proposal is to increase our understanding of hematopoietic (HSC) differentiation with the ultimate goal of improving the therapy of patients with hematological, immunological, and genetic diseases. The proposal is multi-disciplinary clinical HSC transplantation. The proposal contains four Projects and three Cores including an Administrative Core that contains a Pilot Project.
The Specific Aims of the Projects include 1) the regulation of human HSC cell cycle induction, 2) the regulation of HSC differentiation, 3) the role of cytokine receptor interactions in lymphohematopoietic differentiation, 4) an evaluation of HSC transduction by lentivirus vectors, and 5) a clinical gene therapy trial for adenosine deaminase (ADA) vectors. Overall the proposal is an integration of basic/pre-clinical laboratory research and clinical utilization of HSC.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Specialized Center (P50)
Project #
5P50HL054850-07
Application #
6389512
Study Section
Special Emphasis Panel (ZHL1-CSR-C (S1))
Program Officer
Peterson, Charles M
Project Start
1995-09-30
Project End
2005-08-31
Budget Start
2001-09-15
Budget End
2002-08-31
Support Year
7
Fiscal Year
2001
Total Cost
$1,452,683
Indirect Cost

  Institution

Name
Children's Hospital of Los Angeles
Department
Type
DUNS #
094878337
City
Los Angeles
State
CA
Country
United States
Zip Code
90027

  Publications

Candotti, Fabio; Shaw, Kit L; Muul, Linda et al. (2012) Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood 120:3635-46
Rappeport, Joel M; O'Reilly, Richard J; Kapoor, Neena et al. (2010) Hematopoietic stem cell transplantation for severe combined immune deficiency or what the children have taught us. Immunol Allergy Clin North Am 30:17-30
Bauer, Gerhard; Dao, Mo A; Case, Scott S et al. (2008) In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Mol Ther 16:1308-15
Dao, Mo A; Nolta, Jan A (2007) Cytokine and integrin stimulation synergize to promote higher levels of GATA-2, c-myb, and CD34 protein in primary human hematopoietic progenitors from bone marrow. Blood 109:2373-9
Engel, Barbara C; Podsakoff, Greg M; Ireland, Joanna L et al. (2007) Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report. Blood 109:503-6
Hollis, Roger P; Nightingale, Sarah J; Wang, Xiuli et al. (2006) Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon. Exp Hematol 34:1333-43
Hess, David A; Wirthlin, Louisa; Craft, Timothy P et al. (2006) Selection based on CD133 and high aldehyde dehydrogenase activity isolates long-term reconstituting human hematopoietic stem cells. Blood 107:2162-9
Montecino-Rodriguez, Encarnacion; Leathers, Hyosuk; Dorshkind, Kenneth (2006) Identification of a B-1 B cell-specified progenitor. Nat Immunol 7:293-301
Buckley, S; Barsky, L; Weinberg, K et al. (2005) In vivo inosine protects alveolar epithelial type 2 cells against hyperoxia-induced DNA damage through MAP kinase signaling. Am J Physiol Lung Cell Mol Physiol 288:L569-75
Podsakoff, Greg M; Engel, Barbara C; Carbonaro, Denise A et al. (2005) Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells. Mol Ther 12:77-86

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