Gene therapy aims to fix sickle cell at its source
NCT ID NCT03282656
First seen Jan 11, 2026 · Last updated May 01, 2026 · Updated 14 times
Summary
This study tests a gene therapy for people with severe sickle cell disease. The approach uses a patient's own blood stem cells, modified to boost fetal hemoglobin, which can reduce sickling. Ten participants will receive the treatment to see if it is safe and can increase fetal hemoglobin levels.
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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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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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