Gene therapy offers hope for kids with rare bone marrow disease
NCT ID NCT04248439
First seen Jan 05, 2026 · Last updated May 21, 2026 · Updated 21 times
Summary
This study tests a gene therapy for children with Fanconi anemia subtype A, a rare genetic condition that leads to bone marrow failure. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and put the corrected cells back into the body. The goal is to restore the bone marrow's ability to make healthy blood cells and prevent the need for a transplant.
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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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Stanford University
Stanford, California, 94304, United States
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University of Minnesota
Minneapolis, Minnesota, 55454, United States
Conditions
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