Beyond transfusions and transplants: genomic innovations rewriting the narrative of thalassemia.

ABSTRACT: Thalassemia is a globally prevalent inherited blood disorder that usually leads to severe complications and even premature death due to impaired hemoglobin synthesis. The conventional treatment approach encompasses a range of interventions, including red blood cell transfusions, iron chelation therapy, splenectomy, and allogeneic hematopoietic stem cell transplantation. However, transfusion-induced iron overload and the limitation of graft matching have emerged as significant clinical impediments. In recent years, with the advent of precision medicine and translational research, the treatment of thalassemia has undergone a paradigm shift toward stem cell gene therapy, gene editing combined with nanodelivery, and pharmacogenomics-guided, personalized treatment regimens. In preclinical and e

SUBMITTER: Shi L 

PROVIDER: S-EPMC12431938 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature