An article published last week reported on groundbreaking work by researchers who have successfully reversed a teenager’s sickle cell anemia with the world’s first gene-editing treatment for the disease.
Sickle cell disease is a condition in which the body’s bone marrow creates defective blood cells in the shape of a sickle, rather than a sphere. These sickle-shaped cells then block blood flow throughout the body causing extreme pain to the patient. Sickle cell disease affects over 100,000 Americans, and many more worldwide.
The subject who received the therapy in question reportedly had so much disease-inflicted damage on his internal organs, that he had to receive a blood transfusion every month while on medication.
Doctors at the Necker Children’s Hospital in Paris achieved these results by first draining the 13-year-old’s bone marrow. They then “infected” the marrow with a virus containing genetic instructions to correctly manufacture healthy red blood cells, instead of the deformed ones that blocked blood flow.
After the insertion of the edited bone marrow into the teenager 15 months ago, the youth was able to stop taking medication and has shown no symptoms of sickle cell disease since.
Specialists are reluctant to call this procedure a “cure” since more tests need to be conducted, however the triumph has raised hope for millions of people suffering from the same disease.
(Source: New England Journal of Medicine)