A number of recent headlines imply a recent case study just published…proves that gene therapy has cured sickle cell disease—a genetic disorder that incurs tremendous pain, suffering and diminished life expectancy.
Due to such limited progress in management of this condition, this team of researchers…took samples from the bone marrow of a patient with severe disease…Using a lentiviral vector, they transferred an anti-sickling gene into the patient’s stem cells…which get put back into the patient in the hope they will multiply and replace the cells made with the defective gene.
[T]he team concludes their patient “had complete clinical remission with correction of hemolysis and biologic hallmarks of the disease.” Furthermore, after fifteen months, the antisickling protein remained high at approximately 50% and the patient had no crises or hospitalizations. Before, the patient required regular transfusions.
ACSH’s Senior Fellow in Molecular Biology, Dr. Julianna LeMieux, puts the promise of gene therapy into even greater context for this and other disease entities: “This is an incredibly promising result, even with the obvious caveat that it is only one person. Sickle Cell is a disease that is ripe for genetic advances…[T]his one success story is incredibly encouraging for the sickle cell community and for moving the field of curing diseases using genetic editing forward.”
[The study can be found here.]
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