A limitation in FA research is the absence of a live model that displays key features of the disease. Researchers at OHSU will create the first large animal model of FA to test potential therapies for people with FA.
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Fanconi anemia patients have a very high predisposition to head, neck, and anogenital squamous cell carcinoma (SCC). To understand how these cancers develop, Dr. Smogorzewska and team are cataloging genetic changes that are present in the cancers. They are using...
There is a great deal of excitement about the potential of CRISPR genome editing to treat disorders such as Fanconi anemia. But the mutations that cause FA also reduce the effectiveness of genome editing. Dr. Corn and colleagues will search...
Grant description: Beyond surgical resection, there is no effective therapy nowadays to treat FA patients that develop solid tumors, so a drug able to kill tumor cells but nontoxic for FA patients is of critical relevance. Gefitinib and afatinib candidates...
Oral cancer in Fanconi patients is a severe disease requiring innovative treatment and prevention measures. The potential use of FDA-approved diabetes drugs is a practical strategy for Fanconi patients for oral cancer prevention or treatment studies, once these preliminary studies...
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...