Doctors at the Necker Children’s Hospital in Paris discovered that sickle cell anemia could be cured by gene therapy. They made this finding when a boy who was suffering from this terrible disease was completely cured after being treated in a genetic engineering therapy.
The numbers of children who are born with sickle cell disease are tremendously high. All over the world, 275,000 children suffer from this illness and, only in United States, 100,000 sick children are born every year. This disease is spread mostly among African Americans. One in every 365 children suffers from the disease from birth.
The disease is inherited genetically and it affects blood, namely the hemoglobin quantity in the red blood cells is affected and blood can clog in veins and organs. This leads to a shorter life span in sickle cell disease patients. Dr. Grace Onimoe, member of the American Sickle Cell Anemia Association, declared that their life is reduced by several decades.
The doctors involved in the treatment of the cured patient explained how this was possible. Since he was 13, the patient has received treatment for 15 months and he now shows no sign of the disease. His blood cells are perfectly normal.
His pains stopped when he started receiving the treatment. He experienced no complications and transfusions are no longer needed. He feels like a normal child right now.
Phillippe Leboulch, the doctor responsible with administering the treatment, explains that sickle cell anemia is an inherited genetic disorder that causes the mutation of hemoglobin that leads to the deformation of the cells’ shape. This is why the blood forms clogs.
He also explained the treatment process. They extracted bone marrow from the patient, out of which they harvested stem cells and used them to alter the genetic code and make normal hemoglobin.
They did this after a short session of chemotherapy where they fixed the affected stem cells they had taken from the bone marrow. After this procedure, the patient showed an improvement in his conditions, and he no longer needed blood transfusions.
The only long-term treatment method available was bone marrow transplant, but such a treatment is not eligible for all patients, might cause prolonged pain and require extensive blood transfusions. Now, thanks to gene therapy, doctors no longer need a donor and can take stem cells directly from the patients’ bone marrow.
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