One-Shot CRISPR treatment aims to free patients from lifelong blood transfusions
NCT ID NCT03655678
Summary
This study tested a one-time treatment using the gene-editing tool CRISPR to try to help people with severe thalassemia. Doctors took a patient's own blood stem cells, edited a gene in the lab, and then gave them back. The main goal was to see if this single treatment could allow patients to stop needing regular blood transfusions for at least a year and be safe.
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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
Conditions
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