How does gene therapy cure sickle cell disease for patients like Major?

Gene therapy corrects the genetic defect in a patient's own blood cells, eliminating the cause of sickle cell disease.

Where did the first pediatric gene therapy for sickle cell occur in Florida?

The first pediatric gene therapy for sickle cell in Florida was administered at Holtz Children's Hospital.

What are the benefits of gene therapy for sickle cell compared to bone marrow transplants?

Gene therapy uses a patient's own cells and is considered safer and less complex than bone marrow transplants requiring donors.

Home / Health / Gene Therapy Cures Teen's Sickle Cell Pain

Gene Therapy Cures Teen's Sickle Cell Pain

16 Dec, 2025

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Summary

A 19-year-old is essentially cured of sickle cell disease with gene therapy.
Gene therapy genetically manipulates a patient's own cells to correct defects.
The groundbreaking treatment offers freedom from daily pain and fatigue.

Gene Therapy Cures Teen's Sickle Cell Pain

A South Florida teenager has experienced a life transformation, becoming one of the first pediatric patients in Florida to receive successful gene therapy for sickle cell disease. This innovative treatment offers a potential cure for the debilitating inherited blood disorder, which previously caused daily pain and fatigue for the 19-year-old. Doctors at Holtz Children's Hospital utilized gene therapy to genetically alter the patient's own cells, correcting the defect responsible for sickle cell.

Unlike traditional bone marrow transplants, which require donors and carry significant risks, this gene therapy approach is considered safer and more effective. The patient underwent chemotherapy and weeks of isolation for the procedure, but the results have been profound. His parents expressed immense relief and joy, noting their son has gone from barely existing to truly living, now able to socialize and plan for the future.