Cyclophosphamide promotes engraftment of gene-modified cells in a mouse model of Fanconi anemia without causing cytogenetic abnormalities

A major hurdle for hematopoietic stem cell (HSC) gene therapy for inherited bone marrow disorders, including Fanconi anemia (FA), is adequate engraftment of gene-modified cells. A phenotypic defect in DNA repair renders FA patients sensitive to alkylating agents such as cyclophosphamide (Cy); howeve...

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Hauptverfasser: Adair, Jennifer E., Zhao, Xin, Chien, Sylvia, Fang, Min, Wohlfahrt, Martin E., Trobridge, Grant D., Taylor, Jason A., Beard, Brian C., Kiem, Hans-Peter, Becker, Pamela S.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2012
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3650894/
https://ncbi.nlm.nih.gov/pubmed/22660274
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00109-012-0905-0
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