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Gene therapy for monogenic disorders of the bone marrow

5 Jun 2015

Abstract excerpt

Ex-vivo gene transfer of autologous haematopoietic stem cells in patients with monogenic diseases of the bone marrow has emerged as a new therapeutic approach, mainly in patients lacking a suitable donor for transplant. The encouraging results of initial clinical trials of gene therapy for primary immunodeficiencies were tempered by the occurrence of genotoxicity in a number of patients. Over the last decade,...

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PMID: 26044877
DOI: 10.1111/bjh.13520

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