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Bone marrow mesenchymal cells for haemophilia A gene therapy using retroviral vectors with modified long‐terminal repeats

1 Jan 2003

Abstract excerpt

Bone marrow (BM) cells are attractive target cells for ex vivo gene therapy of genetic diseases, including haemophilia A. However, BM-derived haematopoietic stem/progenitor cells (HSCs) transduced with factor VIII (FVIII) retroviral vectors, failed to express FVIII in vivo. To overcome the limitations of HSCs for haemophilia gene therapy, BM-derived mesenchymal cells were explored as alternative target cells. The...

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PMID: 12558785
DOI: 10.1046/j.1365-2516.2003.00709.x

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