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.
Directory
David Liu
PhD
David R. Liu is the Richard Merkin Professor and director of the Merkin Institute of Transformative Technologies in Healthcare, vice-chair of the faculty at the Broad Institute of MIT and Harvard, the Thomas Dudley Cabot Professor of the Natural Sciences at Harvard University, and a Howard Hughes Medical Institute (HHMI) investigator. Liu’s research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. His major research interests include the engineering, evolution, and in vivo delivery of genome editing proteins such as base editors and prime editors to study and treat genetic diseases; the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE); and the discovery of bioactive synthetic small molecules and synthetic polymers using DNA-templated organic synthesis and DNA-encoded libraries.
Base editing—the first general method to perform precision gene editing without double-stranded breaks, and a Science 2017 Breakthrough of the Year finalist—as well as prime editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. These technologies are used by thousands of labs around the world and have enabled the study and treatment of genetic diseases. Five base editing clinical trials are already underway to treat leukemia, hypercholesterolemia, sickle-cell disease, and beta-thalassemia, and the first clinical benefit of a base edited therapeutic in a T-cell leukemia trial has been reported.
FA Research Projects
David Liu
The Latest
News & Events
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.