
A team of international scientists have successfully controlled the spread of a rare almost always fatal brain illness called X-linked adrenoleukodystrophy (ALD) with the use of a new kind of gene therapy. The experimental gene therapy involved the use of a defunct version of the AIDS virus.
The X-ALD is form of a deadly but rare genetic brain disease that causes damage to the insulating membrane surrounding nerve cells in the brain. The onset of this illness comes with the patients showing extreme behavioral changes, such as abnormal withdrawal or aggression. Among other symptoms, a few prominent ones include, poor memory, visual loss, learning disabilities, seizures, etc.
The team of scientists conducted the surgery on two young boys who were diagnosed with X-ALD. The treatment included the use of a deactivated human immunodeficiency virus (HIV), that had been modified to deliver healthy and working genes to the patients’ systems. The delivery of the genes effectively halted the spread of the disease and is being hailed as a medical breakthrough. This is first time that scientists have been able to deliver gene therapy via a HIV delivery module.
Although, bone marrow transplant is a yet another means of controlling the disease, but finding a matching bone marrow donor is a time intensive effort and the transplant itself is considered to be an extremely risky surgery. As of now, the two boys, who have been given the gene therapy are said to be responding very well to the treatment.
Via Reuters.
Posted by Rajeev Saxena on November 6, 2009 in Health & Medicine, Sci + Tech · 0 Comment