Sickle cell gene therapy shows promise in small early trial
NCT ID NCT02247843
First seen Jan 11, 2026 · Last updated May 21, 2026 · Updated 14 times
Summary
This early-phase study tested a gene therapy for adults with severe sickle cell disease. Doctors took stem cells from each patient, added a working gene to help produce healthy red blood cells, and gave them back. The main goals were to check safety and see if the treatment could reduce sickle cell complications. Only 4 people took part, so results are very preliminary.
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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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Contacts and locations
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Locations
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095, United States
Conditions
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