A Gene Therapy, Successful In Beta-Thalassemia, Might Lead To Treatments For Other Genetic Disorders

A Gene Therapy, Successful In Beta-Thalassemia, Might Lead To Treatments For Other Genetic Disorders
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French researchers presented the positive results of a gene therapy obtained in patients with a very common and often fatal genetic disorder of the blood- beta-thalassemia. The therapy consisted of genetically modifying blood stem cells. The encouraging results of this treatment were presented Wednesday in the journal New England Journal of Medicine.

Β-thalassemia, also known as red blood cell disease, is a form of hereditary anemia that affects 200,000 children each year at birth. It is characterized by a mutation carried by the two parent chromosomes 11, which causes the alteration of the beta chain of hemoglobin. Under these conditions, the protein complex of hemoglobin is not formed properly and the transport of oxygen is then impaired.

From a clinical point of view, without treatment, the disease is often fatal before the age of 8 years.

In short, beta-thalassemia is a disorder where the beta-globin gene is mutated and does not work properly or not at all or almost, thus, preventing the production of hemoglobin. In its most serious form, it makes patients dependent on a monthly blood transfusion just to keep themselves alive.

New gene therapy increases the hopes for a future cure of other genetic disorders, besides beta-thalassemia

Scientists are adding genes to replace the missing ones or repair the damaged ones, thanks to a kind of “Trojan horse” that will bring therapeutic DNA inside the body.

“For beta-thalassemic patients suffering from the severe form and without a compatible marrow donor, with organs still in good condition, blood stem cells are taken, mixed with the vector, frozen and verified that the gene is well entered the DNA. These samples are thawed and placed on a drip to reinject these modified stem cells,” explained Professor Leboulch, one of the researchers

The vector, called LentiGlobin, is, in fact, the element which carries the modified genes which are injected in patients.

According to the scientists, they’ve already applied this method. As result, a young man with beta-thalassemia has been cured by this technique and other 21 showed great recovery from the disease.

Like any genetic disease, repairing the altered gene or adding a missing one would be the only treatment solutions. Now, the successful gene therapy in cases of beta-thalassemia could open the roads to the treatment for other genetic disorders.


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Jeffrey likes to write about health and fitness topics, being a champion fitness instructor in the past.

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