Gene therapy offers new hope for sickle cell patients
NCT ID NCT03964792
First seen Feb 05, 2026 · Last updated Jun 16, 2026 · Updated 23 times
Summary
This early-phase study tested a gene therapy for sickle cell disease in 6 patients aged 12-20. Doctors took the patients' own blood stem cells, added a working gene to help produce healthy red blood cells, and infused them back after chemotherapy. The goal was to see if the treatment is safe and can reduce severe pain crises and other complications.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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Department of Biotherapy, Necker-Enfants Malades Hospital
Paris, 75015, France
Conditions
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