Allogeneic hematopoietic stem cell transplantation is the only curative treatment for patients
with the non-malignant bone marrow failure syndrome called Fanconi anemia (FA). However, early and
late complications associated with this approach underscore the need for alternative treatments. Gene
therapy approaches aiming to correct the genetic defect in the patient’s own hematopoietic stem cells
remain the most promising strategy to overcome FA-associated bone marrow failure. Yet, despite more
than two decades of clinical research, a therapeutic “success” has not yet been achieved. Here we review
the clinical trials conducted to date and highlight the unique features of FA revealed by these studies.
These features render FA the “holy grail” of hematopoietic stem cell gene therapy approaches, and
identify the future steps required to achieve clinical success in this rare disease.
Keywords: Fanconi anemia, Clinical trials, Gene therapy, Bone marrow failure, Stem cells, Viral vectors.
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