Gene therapy shows promise for sickle cell disease in small trial
NCT ID NCT02247843
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase clinical trial tested a gene therapy for adults with severe sickle cell disease. The treatment involved taking the patient's own blood stem cells, modifying them with a harmless virus to carry a healthy gene, and infusing them back after mild chemotherapy. Only 4 people took part, and the main goal was to check safety. Early results suggest the approach may reduce sickle cell complications, but larger studies are needed to confirm.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
gene-modified stem cells (Lenti/βAS3-FB)
What this could lead to
If successful, this could lead to a one-time treatment that reduces or eliminates sickle cell disease symptoms without needing a donor.
What could go wrong
This was a very small early-phase trial with only 4 participants. Long-term safety and effectiveness are not yet proven, and there are risks from the chemotherapy used before the transplant.
Disclaimer
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the original study
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095, United States