Article
Genome Editing for the β-Hemoglobinopathies.
Advances in experimental medicine and biology - 1 Jan 2017
Porteus Matthew H
Abstract excerpt
The β-hemoglobinopathies are diverse set of disorders caused by mutations in the β-globin (HBB) gene. Because HBB protein is a critical component (along with α-globin, heme, and iron) of hemoglobin, the molecule essential for oxygen delivery to tissues, mutations in HBB can result in lethal diseases or diseases with multi-organ dysfunction. HBB mutations can be roughly divided into two categories: those that...
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