Article
Thalassemias: from gene to therapy.
Molecular aspects of medicine - 1 Apr 2022
De Simone Giovanna, Quattrocchi Alberto, Mancini Benedetta, di Masi Alessandra, Nervi Clara, Ascenzi Paolo
Abstract excerpt
Thalassemias (α, β, γ, δ, δβ, and εγδβ) are the most common genetic disorders worldwide and constitute a heterogeneous group of hereditary diseases characterized by the deficient synthesis of one or more hemoglobin (Hb) chain(s). This leads to the accumulation of unstable non-thalassemic Hb chains, which precipitate and cause intramedullary destruction of erythroid precursors and premature lysis of red blood...
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