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Article

Bone Marrow Stromal Cells as Targets for Gene Therapy of Hemophilia A

10 Feb 1998

Abstract excerpt

Attempts to develop an ex vivo gene therapy strategy for hemophilia A, using either primary T cells or bone marrow (BM) stem/progenitor cells have been unsuccessful, due to the inability of these cell types to express coagulation factor VIII (FVIII). As an alternative, we evaluated the potential of BM-derived stromal cells which can be readily obtained and expanded in vitro. Human and murine BM stromal cells were...

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Identifiers

PMID: 9508053
DOI: 10.1089/hum.1998.9.3-353

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