A major problem in the care of patients with FA is managing the known risk of leukemia. As techniques for genetic analysis have exponentially improved in recent years, we now have the capability of finding very rare DNA mutations in...
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The goal of this project is to identify drugs that improve DNA integrity and bone marrow stem cell function in FA. The Cantor lab has uncovered defects in so-called DNA replication that lead to small gaps and compromise cell turnover...
The objective of this proposal is to better understand the pathogenesis of head and neck squamous cell carcinomas (HNSCCs) for individuals with FA, in order to find practical and proactive preventive strategies to improve their quality of life. Specifically, this...
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...
While the transplantation of healthy blood-forming stem cells from a healthy related or unrelated donor is the current standard of care for FA patients, this treatment carries a significant risk of morbidity and mortality caused by the toxic preconditioning regimen...
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 relies on...