Naisha Chhabria

From Transfusions to Transformation: Assessing Gene Therapy against Conventional Treatments for Thalassemia

Started Apr. 17, 2025

Abstract or project description

Thalassemia is an autosomal recessive hemoglobinopathy that results in severe anemia and organ damage, affecting up to 400 million people worldwide. Alpha thalassemia major and beta thalassemia major are the most severe forms of thalassemia. Alpha thalassemia major is fatal before birth, while beta thalassemia major leads to fatality during childhood. Conventional treatments for thalassemia have many drawbacks and can only manage the symptoms without curing the disorder. Gene therapy has recently emerged as a promising treatment for thalassemia, which could serve as a lifelong cure. Some gene therapies aim to cure beta thalassemia by inserting a gene construct for β-globin production, and others by gene editing to reactivate fetal hemoglobin (HbF) synthesis. Some of these therapies have even been approved to treat the disorder. This paper evaluates conventional treatments for thalassemia and examines the potential of gene therapy as a new treatment approach.