Healing Power of Gene Therapy Resists Beta-Thalassemia Effectively

Healing power of gene therapy resists beta-thalassemia effectively, as the recent exploratory treatment has proved to be shedding a new light on the life saving therapies performed in hospitals. The international clinicians and investigators have just revealed that a gene therapy on the patients suffering through a blood disorder beta-thalassemia will be safer than previous.


One of the most usually diagnosed disorders can now be treated with a novel gene therapy. According to the findings published on Wednesday in the New England Journal of Medicine, the 15 patients among 22 who were treated with the new gene therapy, found to be capable of restraining and intensely decrease the common blood transfusions required for them to relieve the fatal anemia, without any serious side effects.

The disorder has become one of the most commonly found genetic diseases, as it has been estimated that around 288,000 people are suffering from beta thalassemia across the world, but most general in the Mediterranean countries such as Asia, the Middle East, India, South America and Africa. However, nearly 10,000 to 15,000 people are diagnosed with beta-thalassemia in the United States.

Professor of medicine and Institute Director, study’s leading director, Philippe Leboulch, MD from Women’s Hospital’s Division of Genetics said in a statement that, “It was always our hope to bring our research findings to patients. We have taken our work from the lab, through preclinical models, and past the proof-of-principle stage, and are now able to gauge its effectiveness in patients with this disease. It is immensely gratifying.”