CRISPR gene therapy aims to free kids from lifelong blood transfusions
NCT ID NCT05356195
First seen Oct 31, 2025 · Last updated Jun 17, 2026 · Updated 31 times
Summary
This study tests a one-time gene therapy (CTX001) in 16 children with transfusion-dependent β-thalassemia, a genetic blood disorder requiring regular red blood cell transfusions. The treatment uses the patient's own blood stem cells, edited with CRISPR-Cas9, to produce healthy red blood cells. The main goal is to see if children can stop needing transfusions for at least 12 months.
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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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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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