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    What is Fanconi Anemia?

    Fanconi anemia is a genetic disease that mainly affects the bone marrow, resulting in decreased production of all types of blood cells. People with this condition may have bone marrow failure, physical abnormalities, organ defects, and an increased risk of certain cancers.

 

Research Strategies

 

Understanding who is wrong with Fanconi anemia patient cells

Research in Dr. Cantor’s lab is focused on identifying what is wrong with Fanconi anemia patient cells. Fanconi anemia patient cells are very sensitive to agents that interfere with DNA replication. Even in the absence of exogenous genotoxic stress, Fanconi anemia cells have abnormally elevated DNA damage response (DDR). In patients, this constitutively active DDR is most prominent in rapidly dividing tissue and correlates with bone marrow failure. Understanding the underlying cause of the overactive DDR in Fanconi anemia cells could provide clues for therapeutic intervention, to mitigate bone marrow failure or prevent malignancy in Fanconi anemia patients.

 

Fanconi Anemia Investigator

Sharon Cantor, PhD

Sharon Cantor, PhD

Associate professor, molecular, cell and cancer biology

Cantor Lab

Clinical Trials

At the Li Weibo Institute for Rare Diseases Research, our researchers and clinicians are dedicated to finding new treatment options for patients. Our clinical trials program is another way we continue to advance treatments and make new discoveries. If you or someone you know may be interested in a clinical trial, please subscribe for updates and you will be notified when we open up a new trial.