Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia

LUW Müller, DA Williams - Mutation Research/Fundamental and Molecular …, 2009 - Elsevier
Fanconi anemia is a rare bone marrow failure and cancer predisposition syndrome.
Childhood onset of aplastic anemia is one of the hallmarks of this condition. Supportive
therapy in the form of blood products, androgens, and hematopoietic growth factors may
boost blood counts temporarily. However, allogeneic hematopoietic stem cell transplantation
(HSCT) currently remains the only curative treatment option for the hematologic
manifestations of Fanconi anemia (FA). Here we review current clinical and pre-clinical …