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...
Supported Research
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As a monogenic blood disorder with potential survival disadvantage, Fanconi anemia has long been considered an attractive target for conventional gene therapy but success has been elusive. Consequently, there has been increasing interest in developing techniques to catalyze correction of the disease-causing mutation at the...
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My lab has developed a rapid and efficacious Cas9-based approach to introduce programmed sequence changes to human cells with ease. This work takes advantage of our discovery that Cas9 is extremely long-lived on its target DNA, yet releases a flap of single stranded DNA after...
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Current therapy for FA is limited to allogeneic stem cell transplantation (SCT), a process associated with significant morbidity and mortality, particularly when an ideally matched donor is not readily available. Therefore, novel therapies that improve or replace SCT are needed. Progress in FA therapy has...
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By virtue of their inability to repair a specific form of DNA damage, persons with Fanconi Anemia (FA) are appreciably more susceptible to development of certain cancers. Oral squamous cell carcinoma (OSCC), which arises from the lining cells of the mouth, occurs at >500 fold...
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The objective of this proposal is to conduct a pilot study of metformin to treat FA. This pilot study will explore whether Metformin is safe and efficacious in improving hematopoiesis in patients with Fanconi Anemia. We will focus on pediatric and young adult patients with...
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Fanconi anemia (FA) patients suffer from progressive bone marrow failure due to the defective hematopoietic stem cells (HSCs) in their bone marrow. The mechanisms of why the HSCs in FA patients are defective remain elusive. Recent studies suggest that DNA damage induced by physiological stress...
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The cause of injury and loss of blood-forming stem cells (Hematopoietic Stem Cells, HSC) in Fanconi anemia is due to abnormal repair of a specific type of DNA damage in which two DNA strands get permanently knotted to each other. This type of injury probably...
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As a monogenic blood disorder with potential survival disadvantage, Fanconi anemia has long been considered an attractive target for conventional gene therapy but success has been elusive. Consequently, there has been increasing interest in developing techniques to catalyze correction of the disease-causing mutation at the...
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This is a training grant for a medical geneticist from Mexico City, who will develop a registry and subsequently a cohort of patients with FA who reside in Mexico. The project addresses one of the research priorities of the FARF:
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News & Events
FCF has committed $631,164 to three projects addressing critical gaps in FA cancer research. The work spans different stages, from exploring oral gene therapy and screening drugs for FA-specific safety to analyzing clinical experiences that could inform future care. These projects are building the knowledge needed to pursue safer cancer options for people with FA.
Living with Fanconi anemia, or caring for someone with FA, brings ongoing uncertainty, complex medical experiences and emotional strain. These experiences affect more than physical health. They can also shape mental health, relationships, daily routines and a person’s sense of safety and connection. The Fanconi Cancer Foundation is pleased to introduce the Mental Health and Wellbeing Toolkit, a new resource offering practical tools and trusted guidance for people living with FA and those who care for them.
Our son Blake is 14 years old and eight years post bone marrow transplant. While we celebrate milestones, it’s important to remember that the transplant did not cure his Fanconi anemia. It gave him something incredibly valuable, more time, but FA is still part of his life.