The inability to transduce a sufficient fraction of the stem cell pool is a major limitation of gene therapy for sickle cell disease, a painful and debilitating blood disorder that afflicts about one in every 1000 Americans of African descent. In this project we are identifying the optimal conditions for mobilizing hemopoietic stem cells from the bone marrow into the peripheral blood so the greatest number of stem cells can be harvested. We are also investigating optimal conditions for transduction of these cells ex vivo to correct the genetic defect in the sickle cell or in cells from other hemoglobin diseases. We have found that the stem cells from the different species of nonhuman primates vary in their ability to be transduced with our retroviral constructs.

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
Primate Research Center Grants (P51)
Project #
5P51RR000166-38
Application #
6116379
Study Section
Project Start
1999-05-01
Project End
2000-04-30
Budget Start
1998-10-01
Budget End
1999-09-30
Support Year
38
Fiscal Year
1999
Total Cost
Indirect Cost
Name
University of Washington
Department
Type
DUNS #
135646524
City
Seattle
State
WA
Country
United States
Zip Code
98195
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