Grant description: Persons with FA, as well as their family members, voice concern about being underweight and unable to build muscle mass. These concerns have an obvious physical impact, not only on the person's appearance to others and themselves and...
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FA confers a high risk of developing leukemia at a young age, and patients with FA who develop leukemia are difficult to treat with have poor long-term outcomes. Leukemia develops as a consequence of the acquisition of genetic mutations within...
Head and neck cancer is a major issue for the FA population. There is no suitable preclinical model to study potential strategies for preventing or delaying squamous cell carcinoma in FA. Using a cancer-prone mouse model developed by a lab...
Grant description: Treatment of blood complications in FA kids using bone marrow transplantation has seen great improvement over the past decade. However, after a successful bone marrow transplantation, those kids affected with FA must now face nonblood malignancies in subsequence...
Grant description Bone marrow transplantation has been used for 60+ years to treat >1,000,000 patients suffering from many types of blood or immune diseases, and this therapy is the best current treatment for FA patients experiencing blood problems. This procedure...
Fanconi Anemia (FA) is characterized by fragile bone marrow and the inability to repair DNA damage which accumulates in repopulating stem cells, leading to marrow failure. Current treatments often involve bone marrow transplantation however the majority of patients will not...