New FA Therapies

New Fanconi Anemia Therapies

In recent years, the Seattle area has attracted many of the world’s brightest minds in medicine and science. They include specialists in computer science, molecular biology, cancer, inherited diseases, infectious disease, immunology, and genomics.

Today, many of these doctors and scientists work at nearby research centers such as the Hutchinson Center, Seattle Children’s Research Institute, UW Program in Molecular and Cell Biology, UW Institute for Stem Cell and Regenerative Medicine, UW Markey Molecular Medicine Center, Seattle Biomed, and the Northwest Genome Engineering Center.

Increasingly, researchers at these Seattle-based biomedical groups are collaborating with each other and with our FA experts to better understand FA disease pathways and to create new targeted FA treatments.

We benefit from our proximity to this vibrant intersection of computing, biology, medicine, and genome engineering. New ideas emerging out of these interactions have helped us focus FA-related research on:

  • Understanding how cells normally repair damaged DNA and how FA disrupts that repair process 
  • Using DNA-targeted agents to correct the FA gene defect within the patient’s own blood cells 
  • Reprogramming a patient’s own skin cells as a new source of gene-corrected FA-free stem cells 
  • Screening for drugs that alter FA disease pathways inside the cell 
  • Developing a rapid screening test for all FA mutations—a test that will speed diagnosis and help families in genetic counseling
  • Measuring the impacts of specific FA cell defects