Wake-up Sleepy Gene

Disorders in hemoglobin (hemoglobinopathies) were the first monogenic diseases to be characterized and remain among the most common and best understood genetic conditions. Moreover, the study of the β-globin locus provides a textbook example of developmental gene regulation. Merlin Crossley discusses recent discoveries in β-globin genes, focusing on the influence of genome editing technologies, including CRISPR-Cas9, and emerging gene therapy approaches. Image curtesy of Merlin Crossley.

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