Article

Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative Medicine

2026-06-08

Abstract excerpt

<h4>Background: </h4> β-haemoglobinopathies, including sickle cell disease and transfusion-dependent β-thalassaemia, are among the most common monogenic disorders worldwide and represent a major global health burden. Conventional treatments, such as blood transfusions, iron chelation, fetal haemoglobin induction, and allogeneic haematopoietic stem cell transplantation, have im-proved outcomes but remain limited by...

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Literature Corpus work: 6022b896-09d6-574f-b3dc-8b734b51e204
DOI: 10.20944/preprints202606.0584.v1

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