Gene therapy for sickle cell disease often isn’t a cure, Elizabeth Tracey reports

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Gene therapy for sickle cell disease has a breathtaking price tag of 2-3 million dollars per person, and for some people who receive it, is doesn’t actually cure the condition. That’s according to Richard Brodsky, a sickle cell expert at Johns Hopkins and one developer of a new technique to treat sickle cell that costs much less and most often does provide a cure.

Brodsky: They no longer refer to gene therapy and gene editing as a cure, they now call it transformative therapy because the crises are markedly decreased, they're not eliminated in many of the patients. Some of these patients still have disease so it's not a cure. When you do myeloablative busulfan for gene therapy and gene editing you are guaranteed to have a patient that is infertile. These are young people. With this regimen we know that it preserves fertility in probably at least half.   :31

Brodsky says both need for transfusion and hospitalization is reduced with the new technique when compared to gene therapy. At Johns Hopkins, I’m Elizabeth Tracey.