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Successful Gene Therapy for Sickle Cell Disease 


Prior to this report, the only curative therapy for sickle-cell disease was allogeneic bone marrow transplant. But few patients have matching donors, and even if a match is found, lifelong immunosuppression is necessary to prevent rejection. Gene therapy is the future. Here’s how it was done.

First, the researchers harvested stem cells from the child’s bone marrow. These stem cells were exposed to a lentivirus vector containing an alternate hemoglobin gene. Now, here is where it gets really clever. You’d expect that they just put the normal hemoglobin A gene in there, but they didn’t. Instead, they inserted a variant of hemoglobin A with a glutamine-threonine amino acid substitution. This variant has anti-sickling properties. In fact, the variant was discovered in some kids who were expected to have sickle-cell disease but never manifested symptoms.
This is fighting fire with fire. Use a mutated hemoglobin A gene to combat a mutated hemoglobin A gene.

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