A novel treatment that uses gene therapy to correct a faulty gene in the stem cells of the recipient. Stem cells are obtained from the patient, grown and “corrected” in a laboratory, and then returned to the patient.
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The 36th Annual Fanconi Anemia (FA) Scientific Symposium and Adult Retreat took place in Charlotte, North Carolina in September 2024, and brought together researchers, clinicians, advocates, individuals with FA and community members from across the globe. This year’s theme, “It Takes...
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Research supported by the Knights has shaped best practices in prevention, early-detection, and treatment strategies for cancer, benefiting people with FA and providing far-reaching benefits for the broader population.