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Gene therapy offers new hope for sickle cell patients

NCT ID NCT03964792

First seen Feb 05, 2026 · Last updated Apr 29, 2026 · Updated 14 times

Summary

This study tested a gene therapy for people with severe sickle cell disease. Doctors took stem cells from the patient's own blood, added a working gene to help produce healthy red blood cells, and put them back. The goal was to see if this approach is safe and can reduce painful crises and other complications. Six patients aged 12 to 20 took part.

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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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Contacts and locations

Locations

  • Department of Biotherapy, Necker-Enfants Malades Hospital

    Paris, 75015, France

Conditions

Explore the condition pages connected to this study.