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Scientists Found Therapy For Sickle Cell Disease; World-First Procedure Reversed Teen’s Condition

Update Date: Mar 03, 2017 07:50 AM EST

Scientists may have found a therapy that will address sickle cell disease. In a world-first procedure, a teenager diagnosed with the condition is no longer on any medication.

Sickle cell disease causes the red blood cells, normally found in round shape to look like a sickle. Red blood cells are responsible in carrying oxygen around the body. Deformed red blood cells can block the flow of blood around the body, causing severe pain, organ damage and even death.

Scientists altered the genetic instructions in the teen's bone marrow so that it will produce healthy red blood cells. The world-first procedure was carried out at Necker Children's Hospital in Paris, France.

The French teen with sickle cell disease needed to have his spleen removed and his hips replaced. He visits the hospital every month to have blood transfusion so that the defective blood will be diluted.

Doctors removed the teen's bone marrow and genetically altered it in the laboratory to correct the defect in his DNA that causes sickle cell disease. Bone marrow is a part of the body that produces the blood.

Scientists then use a virus to infect the bone marrow to give new correct instructions. With the new instructions, the bone marrow was then put back into the patient.

Philippe Leboulch, a professor of medicine at the University of Paris, said that after 15 months of therapy, the teen with sickle cell disease has no more sign of the disease. He experiences neither pain nor hospitalization, and does not require blood transfusion as well.

However, Leboulch feels that they need to perform the same therapy to other patients diagnosed with sickle cell so they can propose it as part of the mainstream regimen. Experts from other research groups also considered the therapy significant as it gives back the life to patients afflicted with the disease.

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