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 replacing sick blood-forming stem cells...
Supported Research
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Fanconi anemia (FA) patients are at exceptionally high risk of developing epithelial cancers. We aim to identify features of these cancers that provide new insight into their origins, and better ways to treat these cancers in the context of FA patients. As part of this...
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Individuals with FA have an increased risk of developing anogenital squamous cell carcinoma (cancer of the vulva, cervix, vagina, or anus), but the best and safest way to treat these cancers in individuals with FA is unknown because only a small numbers of cases have...
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As individuals with FA reach adulthood, they have an increased risk of developing cancer, especially head and neck and anogenital squamous cell carcinoma. Cancer treatment options for individuals with FA are limited due to the DNA repair defects associated with the disease. Therefore, there is...
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Through the first systematic evaluation of all known FA genes in a selected group of infertile patients showing mild hematological alterations we expect to diagnose adult-onset FA. Our objective is to diagnose the disease before the appearance of severe complications and to allow preventive interventions...
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Head and neck cancer in adult Fanconi anemia patients is often poorly managed with standard treatments. New drugs that work on controlling the cell cycle and cell division, rather than by damaging the cell’s DNA, may be safer in patients with FA. Researchers have preliminary...
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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 the latest DNA sequencing technologies...
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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 for potential ways to re-activate...
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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 are approved drugs and are...
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Institution: Laboratorio de Citogenetica, Instituto Nacional de Pediatría, Mexico City, México Area of expertise: Medical genetics, dysmorphology. My work: I am a medical geneticist from Mexico City, introduced to the field of Fanconi anemia (FA) by my mentor, Dr. Sara Frias, whom...
At the Fanconi Cancer Foundation (FCF), we understand the critical importance of addressing mental health alongside physical health for individuals with Fanconi anemia (FA) and their family caregivers. Recent research on the mental health challenges faced by adults with FA...
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...