Jacob CornMany #CRISPR therapies for #globinopathies such as #SickleCell and #thalassemia
re-express fetal hemoglobin. But there's a lesser-known globin with many advantages: Much more similar to adult globin, already expressed at low levels throughout adult human life, and used as the main globin in some species. ETH Pioneer Fellow Mandy Boontanrart figured out how to use #geneediting to "fix" the delta globin promoter, with potential applications to treat red cell disorders. https://www.biorxiv.org/content/10.1101/2022.12.19.521003v1
re-express fetal hemoglobin. But there's a lesser-known globin with many advantages: Much more similar to adult globin, already expressed at low levels throughout adult human life, and used as the main globin in some species. ETH Pioneer Fellow Mandy Boontanrart figured out how to use #geneediting to "fix" the delta globin promoter, with potential applications to treat red cell disorders. https://www.biorxiv.org/content/10.1101/2022.12.19.521003v1
