BEIJING, Sept. 16 — A 21-year-old patient, who needed monthly transfusions to survive since 3, has made “remarkable” progress after receiving treatment to correct faulty DNA, media reports said Thursday.

The unnamed patient, suffering from beta-thalassemia, was treated in Paris in 2007 and didn’t need a transfusion in two years, according to a study published in the science journal Nature.

Beta-thalassaemia is an inherited blood disorder that affects the body’s ability to create red blood cells.

The common treatment of the disease is bone marrow transplant, but only a small number of patients have chances to receive this treatment because of difficulties in finding the right type of donor.

A team, led by Philippe Leboulch of Harvard Medical School, used a virus as a “Trojan horse” to deliver a slice of DNA into cells which were corrected for the flawed beta-LCR gene.

“At present, approximately three years post-transplantation, the biological and clinical evolution is remarkable and the patient’s quality of life is good,” the study of the team said.

But researchers sounded a note of caution, saying there was a possibility that the patient could develop leukaemia in the future due to side effects from the gene therapy.

Gene therapy has been used since the late 1990s, offering the hope of blocking or reversing inherited disease.

However, successes so far have been few, limited to single-gene disorders, and carried out only under tightly-controlled lab conditions.

The successes only include six children, suffering from a retinal disease called Leber’s congenital amaurosis, and two adults with a myeloid disorder, a disease of white blood cells. 

On Xinhua Web site: http://news.xinhuanet.com/english2010/health/2010-09/16/c_13514512.htm