title: Cellular therapy for fanconi anemia: the past, present, and future.
abstract: 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.
authors.affiliation: The Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, University of Minnesota Blood and Marrow Transplant Program, Minneapolis, Minnesota, USA. macmi002@umn.edu
authors.forename: Margaret L
authors.initials: ML
authors.lastname: MacMillan
authors.forename: Mark R
authors.initials: MR
authors.lastname: Hughes
authors.forename: Suneet
authors.initials: S
authors.lastname: Agarwal
authors.forename: George Q
authors.initials: GQ
authors.lastname: Daley
mesh_terms: D005199:Fanconi Anemia / Q000175:diagnosis / Q000628:therapy*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000639:trends; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000637:transplantation; D008297:Male; D011247:Pregnancy; D019836:Preimplantation Diagnosis