Gene therapy aims to cure sickle cell disease in pioneering trial
NCT ID NCT03282656
First seen Jan 11, 2026 · Last updated May 26, 2026 · Updated 18 times
Summary
This early-phase study tests a gene therapy for sickle cell disease. The approach uses a patient's own blood stem cells, modified to boost fetal hemoglobin and reduce sickle hemoglobin, potentially curing the condition. Ten participants with severe sickle cell disease will receive the treatment, with a focus on safety and engraftment success.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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UCLA - Mattel Children's Hospital
Los Angeles, California, 90095, United States
Conditions
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