CRISPR gene editing offers hope for thalassemia patients to ditch transfusions
NCT ID NCT03655678
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long they could go without transfusions. The goal was to see if this one-time treatment could control the disease long-term.
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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British Columbia Children's Hospital
Vancouver, Canada
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Columbia University Medical Center
New York, New York, 10032, United States
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Columbia University Medical Center (21+ years)
New York, New York, 10032, United States
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Imperial College Healthcare NHS Trust, Hammersmith Hospital
London, United Kingdom
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Lucile Packard Children's Hospital
Palo Alto, California, 94304, United States
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Ospedale Pediatrico Bambino Gesù, IRCCS
Rome, Italy
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Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine
Regensburg, Germany
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The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers
Nashville, Tennessee, 37203, United States
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The Hospital for Sick Children
Toronto, Canada
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University College London Hospitals NHS Foundation Trust
London, United Kingdom
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University Hospital Tübingen
Tübingen, Germany
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Universitätsklinikum Düsseldorf Hospital Duesseldorf
Düsseldorf, Germany