First step toward gene therapy for rare anemia: can enough stem cells be harvested?
NCT ID NCT07186179
First seen Oct 31, 2025 · Last updated May 07, 2026 · Updated 28 times
Summary
This study is a first step toward making gene therapy possible for Diamond Blackfan anemia. Researchers want to see if patients can produce enough blood stem cells to be collected for future gene correction. About 10 people aged 3 to 30 will receive two medications to move stem cells from bone marrow into the blood. No actual stem cell collection or gene therapy is done in this study.
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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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Study contacts
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Contact
Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Cohen Children's Medical Center
RECRUITINGNew Hyde Park, New York, 11040, United States
Contact
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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