TY - JOUR
T1 - Cellular Therapy for Fanconi Anemia
T2 - The Past, Present, and Future
AU - MacMillan, Margaret L.
AU - Hughes, Mark R.
AU - Agarwal, Suneet
AU - Daley, George Q.
PY - 2011/1
Y1 - 2011/1
N2 - 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.
AB - 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.
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U2 - 10.1016/j.bbmt.2010.11.027
DO - 10.1016/j.bbmt.2010.11.027
M3 - Article
C2 - 21195298
AN - SCOPUS:78650681401
SN - 1083-8791
VL - 17
SP - S109-S114
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 1 SUPPL
ER -