期刊
MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS
卷 668, 期 1-2, 页码 141-149出版社
ELSEVIER SCIENCE BV
DOI: 10.1016/j.mrfmmm.2009.03.010
关键词
Fanconi anemia; HSC; Gene therapy; Bone marrow failure
资金
- St. Baldrick's Foundation
- NIH [R01 HL081499, DK074310]
Fanconi anemia is a rare bone marrow failure and cancer predisposition syndrome. Childhood onset of aplastic anemia is one of the hallmarks of this condition. Supportive therapy in the form of blood products, androgens, and hematopoietic growth factors may boost blood counts temporarily. However, allogeneic hematopoietic stem cell transplantation (HSCT) currently remains the only curative treatment option for the hematologic manifestations of Fanconi anemia (FA). Here we review current clinical and pre-clinical strategies for treating hematopoietic stem cell (HSC) failure, including the experience with mobilizing and collecting CD34+ hematopoietic stem and progenitor cells as target cells for somatic gene therapy, the current state of FA gene therapy trials, and future prospects for cell and gene therapy. (C) 2009 Elsevier B.V. All rights reserved.
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