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

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 fertili...

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Bibliographic Details
Published inBiology of blood and marrow transplantation Vol. 17; no. 1; pp. S109 - S114
Main Authors MacMillan, Margaret L, Hughes, Mark R, Agarwal, Suneet, Daley, George Q
Format Journal Article
LanguageEnglish
Published United States Elsevier Inc 2011
Subjects
Fanconi Anemia - diagnosis
Fanconi Anemia - therapy
Female
Hematology, Oncology and Palliative Medicine
Hematopoietic Stem Cell Transplantation - methods
Hematopoietic Stem Cell Transplantation - trends
Humans
Induced Pluripotent Stem Cells - transplantation
Male
Pregnancy
Preimplantation Diagnosis
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Summary: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.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
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ObjectType-Review-1
ISSN:1083-8791
1523-6536
DOI:10.1016/j.bbmt.2010.11.027