Key PointsThe RBP regulates fetal hemoglobin directly through posttranscriptional control and indirectly via BCL11A.IGF2BP1 binding to a stop codon–proxima

Immunodeficiency chronic granulomatous disease (CGD) is a rare disorder characterized by defects in a multiprotein complex that generates superoxide in phagocytic cells.1 Recurrent infections and f...

Nature Reviews Drug Discovery - CRISPR-based technologies provide a diverse set of tools to correct pathogenic mutations. This Review describes the promise and the challenges of genome editing...

Nature - Custom CRISPR—Cas9 PAM variants via scalable engineering and machine learning

Metformin could reduce people’s risk of certain age-associated blood cancers, but more work is needed to identify who is likely to benefit.