Posted on Apr 23, 2021, 3:00 PMUpdated on Apr 23, 2021, 3:09 PM
You may have heard of CRISPR-Cas9, these molecular scissors capable of cutting DNA and making changes to it. They earned the American Jennifer Doudna and the French Emmanuelle Charpentier the 2020 Nobel Prize in Chemistry. Barely ten years after the first articles published by these two researchers on the subject, CRISPR-Cas9 is being used in numerous clinical trials, with remarkable preliminary success.
Among the first targets of these new tools are two genetic blood diseases known as “sickle cell anemia” and “beta thalassemia”. Treatment involves taking stem cells from patients’ blood and genetically modifying them in the laboratory to produce functioning hemoglobin. The modified cells are then reinjected into the patient. More than a year after their treatment, patients, whose lives were punctuated by hospital stays and blood transfusions, now no longer need them.
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