Many have proposed that lentiviral vector (LV)-mediated hematopoietic stem cell (HSC) gene therapy may constitute a new safe and efficient approach for the treatment/prevention of the bone marrow failure (BMF) characteristic of FA patients. To further improve the possibilities of...
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Dry and moist skin (e.g., in the mouth) plays a critical role in maintaining a barrier against environmental insults and cancer. Based on new data, this team believes that individuals with FA may have an impaired barrier and this may...
Dr. Tong recently discovered a novel gene called “LNK” that regulates bone marrow cell survival and growth. She found that loss of LNK in a mouse model of FA restores normal bone marrow functions and increases stem cell longevity. This...
Fanconi anemia (FA) patients are at high risk of developing head and neck cancer and have limited options for treatment due to their sensitivity to chemotherapy and radiation. We have recently found that FA model mice with mutations in certain...
Grant description: This project seeks to identify cellular pathways that could be targeted to treat or prevent cancer in FA. The investigators take a novel approach using a high-resolution CRISPR knockout library to screen for fitness genes and FANCA-specific cancer...
A major limitation in FA research is the absence of an animal model that faithfully recapitulates the clinical features of this disease in humans. While mice have the characteristic DNA repair defects, they do not spontaneously develop the progressive anemia...