Oxymetholone Therapy of Fanconi Anemia Induces Hematopoietic Stem Cell Cycling By Suppressing Osteopontin Transcription

Oxymetholone Therapy of Fanconi Anemia Suppresses Osteopontin Transcription and Induces Hematopoietic Stem Cell Cycling

Androgens are widely used for treating Fanconi anemia (FA) and other human bone marrow failure syndromes, but their mode of action remains incompletely understood. Aged Fancd2(-/-) mice were used to assess the therapeutic efficacy of oxymetholone (OXM) and its mechanism of action. Eighteen-month-old Fancd2(-/-) mice recapitulated key human FA phenotypes, including reduced bone marrow cellularit...

Is hematopoietic stem cell homing deficient in Fanconi anemia?

We read with interest the article by Zhang and colleagues comparing progenitor colony formation, chimerism after xenotransplanta-tion into immunodeficient mice, and adhesive properties of whole bone marrow (WBM) cells from Fanconi anemia (FA) patients and healthy donors. 1 We applaud the study of this important topic, but would caution that assays of clonogenicity and hematopoietic repopulation...

allogenic hematopoietic stem cell transplantation from related donors in fanconi anemia

introduction: allogeneic hematopoietic cell transplantation (hsct) is the only therapeutic modality capable of correcting the hematologic manifestations of fanconi anemia (fa). the development  of well  tolerated,  immunosuppressive  conditioning  regimens  for fa patients  undergoing hsct has proven to be a rather challenging task for hematologists. methods: we analyzed the outcome of 30 fa pa...

Oxymetholone therapy in aplastic anemia.

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

Survival rates after allogeneic hematopoietic cell transplantation (HCT) for Fanconi anemia (FA) have increased dramatically since 2000. However, the use of autologous stem cell gene therapy, whereby the patient's own blood stem cells are modified to express the wild-type gene product, could potentially avoid the early and late complications of allogeneic HCT. Over the last decades, gene therap...