Using CRISPR-Cas9 to repair the HBD promoter leads to increased production of hemoglobin HbA2.
Summary
Βeta-hemoglobinopathies, such as sickle cell disease and β-thalassemia, are genetic diseases caused by gene mutations that lead to dysfunctional β-globin subunits in the adult hemoglobin. A recent scientific effort by the Corn Lab at ETHZ (https://www.cornlab.com) used CRISPR-Cas9 to edit transcriptional elements, causing increased amount of adult hemoglobin A2 produced by the cells, a result that could lead to the development of new treatment strategies for β-hemoglobinopathies.
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