Gene therapy breakthrough aims to stop sickle cell pain crises
NCT ID NCT05353647
First seen May 12, 2026 · Last updated May 24, 2026 · Updated 2 times
Summary
This study tests a gene therapy that modifies a patient's own blood stem cells to increase fetal hemoglobin, a healthy type that prevents sickling. The goal is to reduce or eliminate severe pain crises in people with sickle cell disease. About 25 participants aged 13 to 40 with severe disease will receive the treatment and be followed for 2 years. The approach is similar to a bone marrow transplant but uses the patient's own cells, avoiding donor-related risks.
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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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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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Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, 30322, United States
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Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
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Dana-Farber Cancer Institute/Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
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Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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UC Davis Medical Center
Sacramento, California, 95817, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
Conditions
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