In this episode of The Future of Medicine, we welcome Alexis Thompson, MD, MPH, pediatric hematologist and internationally recognized expert on sickle cell disease whose work helped lead to the first approved gene therapies for this serious condition.

Dr. Thompson reflects on the extraordinary transformation of sickle cell care over the course of her career. From the early days of newborn screening and simple interventions like penicillin to today’s breakthrough gene therapies, the field has experienced what she calls “an amazing arc” of progress.

Dr. Thompson also walks through the remarkable science behind gene therapy for sickle cell disease, and how CRISPR gene editing and lentiviral vectors (modified viruses such as HIV that are engineered to safely deliver genes) are being used to modify patients’ own stem cells, allowing them to produce healthy red blood cells and dramatically reduce the complications of the disease.

Looking ahead, Dr. Thompson shares her perspective on the next frontier of genetic medicine: including efforts to make gene therapies safer, more scalable, and accessible to patients around the world.

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