Publication by the S.M. Siegner et al.: Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells.

In Fanconi Anemia (FA), a genetic disorder associated with bone marrow failure and cancer predisposition, DNA repair is defective, and consequently homology directed repair (HDR)-based editing is impaired in FA. We show that base editing approach is a feasible approach for efficient restoration of function in FA patient hematopoietic stem and progenitor cells.