A French boy experienced an unprecedented recovery from the sickle cell disease thanks to genetic engineering.
Genetic engineering managed to show the potential of curing several complex diseases by altering the makeup of cells in one’s system. One of its recent successes regards the sickle-cell disease. This illness usually is inherited from parents and follows the victims throughout their entire life. The life expectancy for such patients is between 40 and 60 in the United States. However, their quality of life has much to suffer because of pain. The blood cells have the shape of a sickle which can cause obstructions of the blood flow. Thus, the body doesn’t receive enough oxygen to all its tissues.
Until recently, there were no signs of a cure for the red blood cell disorder. However, two years ago a teen boy from France accepted a gene therapy treatment for her sickle cell disease. The results of the procedure were published this Thursday in the New England Journal of Medicine. The patient is now better and is no longer taking medicines for his illness. Moreover, he stopped receiving blood transfusions that used to reduce some of the effects of the disease. The reason behind all these is that half of his red blood cells are no longer sickle cells.
However, the study lacks depth as it documented how genetic engineering cured one single patient. Bluebird Bio was the biotech company that funded the clinical trial. The French boy was the only one out of a total of seven whose response to treatment was reported. Some of the others didn’t experience the same healing process as the boy. On the other hand, scientists consider this to be a matter of adjusting the gene therapy for each patient. Moreover, there are some chances that the French boy is going to suffer from other blood flow blockages in the future.
For the new cutting-edge procedure, scientists started feeding the bone marrow stem cells of the boy with genetic engineered versions of beta-goblins. These are proteins invested with the capacity to produce healthy hemoglobin. The procedure was repeated until the boy started to produce normal hemoglobin on his own. In just a matter of months, the number of blood cells with normal shapes increased gradually. In December 2016, the boy had more than half of his blood cells healthy.
However, researchers are aware of the fact that they have just scratched the surface of what means genetic engineering. This field needs more years and extensive funds to show massive progress. Scientists hope that one day, their research will help millions of patients and not just one.
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