One-Shot gene therapy tested to fix bleeding disorder
NCT ID NCT00979238
Summary
This early-stage study is testing the safety of a one-time gene therapy for adults with severe hemophilia B. Researchers are using 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 clotting protein. The study is carefully monitoring a small group of participants to see if this approach is safe and well-tolerated.
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Contacts and locations
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Locations
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Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213, United States
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Katharine Dormandy Haemophilia Centre and Haemostasis Unit, University College of London
London, United Kingdom
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Oregon Health and Science University
Portland, Oregon, 97239, United States
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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
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University of Kentucky
Lexington, Kentucky, 40536, United States
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University of Texas Southwestern
Dallas, Texas, 75390-9063, United States
Conditions
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