Article
Fancl-mutant mice reveal central role of monoubiquitination in Fanconi anemia and a model for therapeutic gene editing.
Blood advances - 10 Feb 2026
Liu Lu, Glaser Astrid, Isiaku Abdulsalam I, Fairfax Kirsten, Casolari Debora A, Ristovski Angelina, Murphy Vincent, van Twest Sylvie, Henrikus Sarah S, Heraud-Farlow Jacki, Granger Elissah, Novakovic Stevan, Monks O'Byrne Sophie F, Tsui Vanessa, Conyers Rachel, Gonda Thomas J, Crismani Wayne, D'Andrea Richard J, Heierhorst Jörg, Deans Andrew J
Abstract excerpt
ABSTRACT: Fanconi anemia (FA) is a rare genetic disorder causing the progressive loss of hematopoietic stem cells (HSCs) and bone marrow failure. Most cases result from deficient monoubiquitination of FANCD2 by the FA core complex. However, given that additional functions for the complex have been proposed, it remains unclear whether loss of FANCD2 monoubiquitination is the sole cause of all FA phenotypes. Here,...
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