One-Time gene injection tested to fix bleeding disorder
NCT ID NCT00979238
Summary
This early-stage study tested the safety of a one-time gene therapy for adults with severe Hemophilia B. Researchers used a modified, harmless virus to deliver a working copy of the Factor IX gene directly to the liver, aiming to help the body produce its own missing clotting protein. The goal was to see if this approach was safe and could potentially reduce the need for frequent clotting factor infusions.
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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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Katharine Dormandy Haemophilia Centre and Haemostasis Unit, University College of London
London, United Kingdom
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Scott and White Memorial Hospital
Temple, Texas, 76508, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38119, United States
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Stanford Medical School
Stanford, California, 94305, United States
Conditions
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