Gene Therapy Could End Lifelong Transfusions For Rare Blood Disorder

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. Image by kjpargeter on Freepik

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. For patients, this would mean liberation from weekly or monthly hospital visits, reduced risk of organ damage caused by iron overload, and a vastly improved quality of life. For families and healthcare systems, it could also mark a turning point: replacing decades of chronic care with a permanent solution. That is the promise of a new study showing how gene therapy may restore healthy red blood cell function in patients with thalassemia.

A New Study Bring