Gene therapy may free patients from lifelong blood transfusions
NCT ID NCT04105166
First seen Nov 01, 2025 · Last updated May 25, 2026 · Updated 27 times
Summary
This early-stage trial tested a gene therapy for people with pyruvate kinase deficiency, a rare blood disorder that often requires frequent blood transfusions. Four adults and children received their own blood stem cells modified to carry a working copy of the faulty gene. The main goal was to check safety, but researchers also looked for signs that the treatment could reduce or eliminate the need for transfusions.
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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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Hospital Infantil Universitario Niño Jesús
Madrid, 28009, Spain
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Hospital Universitario Fundación Jiménez Díaz
Madrid, Spain
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Stanford University
Stanford, California, 94304, United States
Conditions
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