CRISPR gene therapy may free kids with thalassemia from lifelong transfusions
NCT ID NCT05356195
First seen Oct 31, 2025 · Last updated May 20, 2026 · Updated 26 times
Summary
This study tests a single dose of CTX001, a gene therapy made from the patient's own blood stem cells edited with CRISPR, in 16 children with transfusion-dependent beta-thalassemia. The goal is to see if it can help them produce enough healthy red blood cells to stop needing regular blood transfusions for at least a year. Participants receive their own edited cells after a short course of chemotherapy to make room for them.
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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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