Therapeutic gene editing in CD 34 + hematopoietic progenitors from Fanconi anemia patients
نویسندگان
چکیده
منابع مشابه
Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients
Gene targeting constitutes a new step in the development of gene therapy for inherited diseases. Although previous studies have shown the feasibility of editing fibroblasts from Fanconi anemia (FA) patients, here we aimed at conducting therapeutic gene editing in clinically relevant cells, such as hematopoietic stem cells (HSCs). In our first experiments, we showed that zinc finger nuclease (ZF...
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We thank O’Neill and colleagues for their comments on our paper.1 We agree that assays of clonogenicity and hematopoietic repopulation are not sufficient for defining homing. However, we argue that CFC (colony-forming cell) homing assay is an acceptable in vivo approach to assess the homing capacity of human hematopoietic progenitor cells in a mouse bone marrow (BM) transplantation model.2,3 We...
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Fanconi anemia (FA) is a rare autosomal recessive disease, characterized by bone marrow failure and cancer predisposition. So far, 8 complementation groups have been identified, although mutations in FANCA account for the disease in the majority of FA patients. In this study we characterized the hematopoietic phenotype of a Fanca knockout mouse model and corrected the main phenotypic characteri...
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The human Lin2CD342 cell population contains a newly defined class of hematopoietic stem cells that reconstitute hematopoiesis in xenogeneic transplantation systems. We therefore developed a culture condition in which these cells were maintained and then acquired CD34 expression and the ability to produce colony-forming cells (CFC) and SCIDrepopulating cells (SRCs). A murine bone marrow stromal...
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Fanconi anemia is a rare, inherited disorder characterized by bone marrow failure, congenital malformations, and cancer susceptibility. The group C Fanconi anemia gene, FAC, identified by expression cloning methods, encodes a protein of unknown function that may be involved in the response to apoptotic stimuli. Hematopoietic progenitor cells from Fac knock-out mice are hypersensitive to IFN-gam...
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ژورنال
عنوان ژورنال: EMBO Molecular Medicine
سال: 2017
ISSN: 1757-4676,1757-4684
DOI: 10.15252/emmm.201707540