Gene therapies can be used to recover a number of diseases today. Now one more gets added to the list. According to the research study made by the doctors at Boston’s Children Hospital, gene therapy can be used to treat Lorenzo’s oil disease in males. It is one of the deadliest disease diagnosed till date.
Lorenzo’s oil disease scientifically called as adrenoleukodystrophy is a rare genetic disorder that usually occurs in those boys who have only one replica of X chromosome. This one copy of X chromosome is the main cause of the disease.
15 out of 17 individuals today who were treated with gene therapy showed no symptoms of adrenoleukodystrophy when examined after a period of two years. A couple in New York, Paul and Liliana Rojas gave birth to two kids, Brian and Brandon.
Both the kids were diagnosed with this rare kind of disease. However, one of two Brian, 7 years of age underwent the treatment of gene therapy whereas Brandon, 10 years of old did not get any treatment as he was diagnosed earlier than Brian and was easily recoverable without any treatment.
The study and its prolific results were published in New England Journal of Medicine. A number of other therapy producers were involved in the study making a valuable contribution to the conference held in Kansas, Missouri.
Leukemia is the only gene therapy treatment that is available in the US. Hence, the scientists are coming up with other useful and affordable treatments. One out of 20,000 boys every year become the victim of this disorder all over the world.
How this disorder affects body’s internal functioning?
Group of proteins is responsible for breaking down of fatty acids during metabolism. Due to the presence of an only single copy of chromosomes, the body resists the production of such proteins that further increases a number of fats in the body thus destructing the muscles, nerves, and brain.
New gene therapy involves removing of stem cells from blood, altering them, injecting a healthy gene and then placing them intravenously back into the body. The therapy will soon roll out to all the Hospitals and health care providers of US.