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Genetic Engineering of Hematopoietic Stem Cells to Improve the Chance for Racial Minorities to Match with allogeneic unrelated Donors

ABSTRACT: Mismatch of HLA adversely impacts the outcome of patients after allogeneic hematopoietic stem-cell transplantation (alloHSCT). This translates into the requirement to identify suitable HLA-matched donors which curtails the chances of recipients, especially those from a racial minority, to successfully undergo alloHSCT. We sought to broaden the existing pool of registered unrelated donors based on analysis that eliminating the expression of the HLA-A increases the chance for finding a donor matched at HLA-B, -C, and -DRB1 regardless of patients’ race. This was achieved using artificial zinc finger nucleases to eliminate expression of HLA-A alleles in HSC while demonstrating the ability of these engineered stem cells to reconstitute hematopoiesis. The loss of HLA-A expression decreases the need to recruit large number of donors to match with potential recipients and has particular impact for patients whose HLA repertoire is under-represented in the current donor pool to find an appropriate HLA-matched donor

ORGANISM(S): Homo sapiens

PROVIDER: GSE67093 | GEO | 2015/03/21

SECONDARY ACCESSION(S): PRJNA278934

REPOSITORIES: GEO

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