Scientists test One-Shot gene therapy to cure devastating blood disease
NCT ID NCT03653247
Summary
This early-stage study tested a one-time treatment for adults with severe sickle cell disease. Doctors collected a patient's own blood stem cells, used gene editing to potentially fix the disease-causing problem, and then transplanted the modified cells back. The main goals were to see if this complex procedure was safe and if it could provide a lasting benefit, potentially curing the disease.
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Contacts and locations
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Locations
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Investigational Site Number 101
Bethesda, Maryland, 20892, United States
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Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
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UCSF Benioff Children's Hospital
Oakland, California, 94609, United States
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University of California Davis Comprehensive Cancer Center
Sacramento, California, 95817, United States
Conditions
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