Gene editing could cure sickle cell and thalassemia in new trial
NCT ID NCT06647979
First seen Jan 06, 2026 · Last updated May 05, 2026 · Updated 26 times
Summary
This study tests a gene therapy that edits a patient's own blood stem cells to produce more fetal hemoglobin, a healthy type that can replace the defective hemoglobin causing sickle cell disease or beta-thalassemia. The treatment involves a stem cell transplant using the patient's own edited cells, avoiding the risk of graft-versus-host disease. The trial aims to see if this approach can safely cure these severe blood disorders in 10 participants aged 13 to 40.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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