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.
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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.Share this article
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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.]The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: Did Gene Therapy Cure Sickle Cell Disease?
