Small study paves way for gene therapy in rare childhood disease
NCT ID NCT02678533
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 22 times
Summary
This early-stage study tested whether a combination of two drugs (G-CSF and plerixafor) can safely collect enough blood stem cells from children with Fanconi anemia for future gene therapy. Only 4 children took part, and the main goal was to see if the cell collection was possible and how well it was tolerated. The results will help design later gene therapy studies.
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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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Hôpital necker-Enfants malades
Paris, PARIS, 75015, France
Conditions
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