Scientists test One-Shot gene fix aiming to cure sickle cell
NCT ID NCT03282656
Summary
This is an early safety study testing a one-time gene therapy that aims to cure sickle cell disease. Doctors collect a patient's own blood stem cells, use a modified virus to insert a corrective gene, and then return the cells after chemotherapy. The goal is to see if this approach is safe and can increase healthy fetal hemoglobin to potentially cure the condition. The study is enrolling up to 10 children and adults (ages 3-40) with severe sickle cell disease.
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Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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UCLA - Mattel Children's Hospital
Los Angeles, California, 90095, United States
Conditions
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