Advances in the treatment of alpha-thalassemia.

Abstract

Alpha (α)-thalassemia represents a group of recessively inherited hemoglobin disorders marked by deficient or absent synthesis of 1 to all 4 of the α-globin genes. Inactivation of 3 α-globin genes--either by deletional or nondeletional mutations--results in hemoglobin H (Hb H) disease. Patients with Hb H disease produce hemoglobin composed of all beta… (More)
DOI: 10.1016/S0268-960X(12)70010-3

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