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- Title
Genetic Correction of Hematopoiesis in Fanconi Anemia: The Case for a Non-HSC-Autonomous Defect.
- Authors
Skinner, Amy M.; Kurre, Peter
- Abstract
In this article, the author discusses the potential mechanisms involved in hematopoietic stem cells (HSCs) in Fanconi anemia (FA). She relates that HSCs are an intuitive high-value target for therapeutic modification by integrating retroviral vectors. Moreover, she infers that the accommodation of the FA HSC phenotype to improve therapeutic strategies will require a systematic analysis and deliberate manipulation of the genetically corrected hematopoietic microenvironment.
- Subjects
RETROVIRUS diseases; HEMATOPOIETIC stem cells; FANCONI'S anemia; GENOTYPE-environment interaction; RETROVIRUSES; THERAPEUTICS; GENE therapy
- Publication
Molecular Therapy, 2009, Vol 17, Issue 8, p1313
- ISSN
1525-0016
- Publication type
Opinion
- DOI
10.1038/mt.2009.138