A CRISPR Alternative for Correcting Mutations That Sensitize Cells to DNA DamageResearchers turned to base editors to correct mutations causing the rare genetic disease Fanconi anemia without inducing double-strand DNA breaks.While this may cure or prevent some of the disease’s complications, stem cell transplantation can cause additional difficulties, including graft-versus-host disease (GvHD) and exacerbated cancer risk.1There is growing interest in applying genome editing technologies like CRISPR-Cas9 to correct Fanconi anemia mutations in patient-derived cells for autologous transplants, in which corrected stem cells are given back to the patient.Because of this, the researchers first examined if CBEs and ABEs would work on non-Fanconi anemia genes in patient-derived cells.“These are very challenging experiments, and more than the experiments, the challenge of [treating] Fanconi anemia is exactly that—the number of cells.”Despite this challenge, the researchers have laid the groundwork for genome editing as a treatment approach in Fanconi anemia, without the need for double-strand DNA breaks.Siegner et al., “Adenine base editing is an efficient approach to restore function in FA patient cells without double-stranded DNA breaks,” 489197, preprint on bioRxiv, 2022."