Supported Research

Correction of Fanconi Anemia Mutations using Digital Genome Engineering

2020 | Research Grant | UNIVERSITY OF MINNESOTA

Amount Funded: $250,000

While the transplantation of healthy blood-forming stem cells from a healthy related or unrelated donor is the current standard of care for FA patients, this treatment carries a significant risk of morbidity and mortality caused by the toxic preconditioning regimen and immunological complications. Our work aims to apply a paradigm – shifting new technology that can efficiently remove the genetic mutation responsible for the FA regardless of FA group, setting the stage for clinical application. These genetically corrected patient cells can then be used for transplantation into the patient and thereby generate a normal immune system.