One-Shot CRISPR treatment aims to cure blood disorder, end lifelong transfusions
NCT ID NCT03655678
Summary
This study tested a one-time treatment for people with severe beta-thalassemia, a genetic blood disorder that requires regular blood transfusions to survive. Doctors used CRISPR gene-editing technology to modify a patient's own blood stem cells, then gave them back as a transplant. The main goal was to see if this single treatment could allow patients to become independent from transfusions for a year or more.
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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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