Gene-Edited stem cells aim to tame sickle cell disease
NCT ID NCT04443907
First seen Jan 11, 2026 · Last updated May 23, 2026 · Updated 20 times
Summary
This early-phase study tested a one-time treatment using the patient's own genetically modified stem cells to boost fetal hemoglobin, which can reduce painful crises and other severe complications of sickle cell disease. Only 4 participants were enrolled before the study was terminated. The goal was to see if the treatment was safe and could lower disease burden, but long-term management would still be needed.
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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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Locations
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Memorial Sloan Kettering Cancer Ctr
New York, New York, 10065, United States
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St Jude Children's Research Hospital
Memphis, Tennessee, 38105-3678, United States
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University of Chicago
Chicago, Illinois, 60637, United States
Conditions
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