Cellular Therapy for Fanconi Anemia: The Past, Present, and Future

Margaret L. MacMillan, Mark R. Hughes, Suneet Agarwal, George Q. Daley

Research output: Contribution to journalArticlepeer-review

15 Scopus citations


Allogeneic hematopoietic cell transplantation (HCT) remains the only proven curative therapy for the hematologic manifestation of Fanconi anemia (FA). Over the past 2 decades, major advances have been made such that transplant outcomes have markedly improved. With the development of in vitro fertilization and preimplantation genetic diagnosis, HLA-matched sibling donor umbilical blood transplantation may be an option for more patients with FA. Recently, the use of pluripotent stem cells has been explored as a novel approach to model the hematopoietic developmental defects in FA, and to provide a potential source of autologous stem cells that can be genetically manipulated and used to generate corrected hematopoietic progenitors.

Original languageEnglish (US)
Pages (from-to)S109-S114
JournalBiology of Blood and Marrow Transplantation
Issue number1 SUPPL
StatePublished - Jan 2011

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