Cancer BiologyLeukemiaPeer-reviewed
SF3B1-mutant mis-splicing of UBA1 confers a targetable therapeutic vulnerability through UBA1 inhibition
Jonas Thier, Sophia Hofmann, Katharina Miria Kirchhof…·Vanessa Lundin
This study identifies UBA1 mis-splicing as a therapeutic target in SF3B1-mutant myelodysplastic syndromes, showing that UBA1 inhibition sensitizes these cells to treatment.
4 citations