Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia

The journal Current Gene Therapy has published an article about gene therapy for Fanconi Anemia. Allogeneic hematopoietic stem cell transplant 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. The full-text article you can find here.