One-Time gene therapy could free kids from lifelong blood transfusions
NCT ID NCT05356195
Summary
This study is testing whether a single treatment using genetically modified stem cells can help children with severe beta-thalassemia become independent from regular blood transfusions. Researchers collect the child's own blood stem cells, use gene editing technology to correct the genetic defect, then return the modified cells to the patient. The main goal is to see if participants can go at least 12 consecutive months without needing any blood transfusions after receiving the treatment.
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Contacts and locations
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Locations
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Great Ormond Street Hospital for Children
London, United Kingdom
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Hospital for Sick Children - Hematology
Toronto, Canada
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IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
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St.Mary's Hospital - Children's Clinical Research Facility
London, United Kingdom
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TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, 37203, United States
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University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
Conditions
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