Abstract
Thalassemias are a very heterogeneous group of hereditary diseases characterized by a decreased or absent synthesis of a globin chain. According to the decreased or absent chain thalassemias are classified into β thalassemia, α thalassemia and other not so frequent types. The β-thalassemia can be a severe transfusion-dependent disease (TDT) requiring periodic scheduled transfusions throughout the patient’s life, as well as less severe forms called intermediate non-transfusion-dependent thalassemia (TNDT) and silent carriers or thalassemic trait, which are asymptomatic. Transfusion and chelation are the two classic pillars on which the treatment of TDT is based. The only curative treatment for the disease was allogeneic transplantation of hematopoietic progenitors, but this is not a therapeutic option for many patients, due to the limitation of finding HLA-identical family donors. We now have a new advanced therapy drug, gene-editing, processed “ex -vivo” in the laboratory, using the CRISPR-Cas 9 technique, which has been approved by the FDA and EMA regulatory agencies for the treatment of TDT. Casgevy it is indicated for patients with TDT, aged 12 years and older, who are candidates for allogeneic transplantation of hematopoietic progenitors but are unable to undergo it due to the lack of an HLA-identical family donor. In this article, we will discuss in more detail this new therapeutic option conditionally approved for patients with TDT that it is postulated as a treatment with potential curative action of the disease.
Published Version
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