NIH R01 · 2025
Developing Innovative Cell and Gene Therapy Strategies to Treat Fanconi Anemia
ABSTRACT/SUMMARY: Fanconi Anemia (FA) is the leading cause of inherited bone marrow failure (BMF) affecting thousands of patients worldwide. While allogenic (allo)-hematopoietic stem cell transplantation (HSCT) outcomes have been improving for FA patients, these treatments currently cause significant morbidity and mortality in part due to the non-specific and genotoxic chemotherapy and/or irradiation conditioning traditionally used pre-transplant. This is especially problematic in FA patients due to their exquisite sensitivity to DNA damage which results in DNA interstrand crosslinks (ICLs) and heightened risk of malignancies in all patients. Our long-term goal is to develop alternative…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.