Gene therapy breakthrough offers hope for sickle cell patients
NCT ID NCT02247843
First seen Jan 11, 2026 · Last updated May 15, 2026 · Updated 13 times
Summary
This early-phase study tested a gene therapy for adults with severe sickle cell disease. Doctors took the patients' own blood stem cells, added a working gene to help produce normal red blood cells, and infused them back. The goal was to see if the treatment is safe and could reduce sickle cell complications. Only 4 people took part, so results are very preliminary.
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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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Locations
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095, United States
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