Researchers from the Necker-Enfants Malades Hospital (Paris) have just published in the New England journal of medicine the results of a world first.
In 2014, these researchers and their colleagues at the Institute of Genetic Diseases “Imagine” collected stem cells from the bone marrow of a teenager with sickle cell anemia (a form of hereditary anemia). Then they “corrected” the genetic defect of these cells by introducing a missing gene (this is called gene therapy) before reintroducing these stem cells into the bone marrow of the young patient.
Two years later, the teenager is in remission and no longer shows any signs of the disease.
The treated teenager hardly ever comes to the hospital again
“This patient came every month to be transfused to avoid having enormous bone pain. He is no longer transfused at all, he hardly ever comes to the hospital again. It gave him the potential to activity which was impossible until now “ explains doctor Jean-Antoine Ribeil from Necker hospital, on the air Europe 1.
The results of this first experiment “confirm the effectiveness of this therapy of the future” emphasize the researchers.
More than 200 babies are born with sickle cell disease each year in France. This genetic disease mainly affects people with dark skin, which is why the DOM is considered a risk area. In these departments, screening is performed on all newborns.
# Sickle cell disease : remission of signs of the disease in the first patient in the world treated with #genetical therapyhttps://t.co/uJ54Ur7moipic.twitter.com/l7OqoqoywI
– AP-HP (@APHP) March 2, 2017
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