First step toward a gene cure for devastating childhood blood disease
NCT ID NCT02678533
Summary
This small, early-stage study tested whether doctors could safely collect enough stem cells from the blood of children with Fanconi anemia. The goal was to see if these cells could then be used in a future gene therapy treatment. Researchers used two drugs to move stem cells from the bone marrow into the bloodstream so they could be collected. The study measured how many cells could be gathered and monitored patients for side effects.
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Contacts and locations
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Locations
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Hôpital necker-Enfants malades
Paris, PARIS, 75015, France
Conditions
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