Creating a stem cell gene therapy for the blood disorder alpha thalassemia | Eva Segura

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Elevator pitch: Eva Segura describes her work to develop a stem cell gene therapy for the genetic blood disorder alpha thalassemia.

Her hope is that by delivering a missing gene directly into patients’ blood stem cells, this one-time therapy could enable the lifelong production of healthy red blood cells.

Segura is a UCLA graduate student in the lab of Donald Kohn, M.D., and a UCLA Broad Stem Cell Research Center trainee.

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