In the field of rare diseases it is extremely difficult to perform clinical trials with new chemical entities without prior information on bioavailability, dose and safety in humans. Drug repurposing is the application of known drugs and compounds to new...
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It has long been recognized that gene-environment interactions influence the clinical course of individuals with FA, yet the underlying mechanisms remain elusive. Drawing on this labs deep expertise in protein folding diseases this project proposes a pragmatic approach to address...
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...
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...