Gene therapy for patients with a severe form of the blood disorder beta-thalassemia could be safe and effective as well as end the need for transfusions, results from the clinical trials show. Beta-thalassemia is a genetic disorder that impairs the body's ability to produce a key component of hemoglobin -- a critical protein in red blood cells that carries oxygen to organs and tissue. People with the most severe forms of beta-thalassemia require monthly blood transfusions, right from their toddler age, to replenish their red blood cell supplies along with iron chelation to remove extra iron from the body.
Frequent transfusions, however, can cause serious complications due to iron toxicity and viral infections. The results, published in New England Journal of Medicine , showed that a one-time treatment with the gene therapy known as LentiGlobin BB305 vector reduced or eliminated the…
