Novel use for selective inhibitors of nuclear export in β-thalassemia: block of HSP70 export from the nucleus

摘要

Thalassemia is the most prevalent monogenic recessive hereditary blood disorder, with around 300,000 children affected at birth worldwide.1 Imbalance between a- and non-a-globin chains results in premature death of differentiating erythroid precursors (erythroblasts), because as a-globin tetramers accumulate and precipitate, they form inclusion bodies that cause oxidative membrane damage and destruction by apoptosis.