Gene Therapies Could Transform the Treatment of Sickle Cell Disease

December 4, 2018

(The Washington Post) – Esrick and colleagues focused on trying to boost levels of a normally functioning fetal form of hemoglobin that is typically shut off after birth. They removed blood stem cells from Johnson and altered them in the laboratory, using a virus to insert a molecule that flips a genetic switch to turn fetal hemoglobin back on. They gave Johnson a form of chemotherapy and then reinfused him with the altered cells. At six months out, they found no sickle cells in his blood, and he has not needed any more transfusions. They plan to give the therapy to the next patient in February, followed by another in March.

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