Gene editing could cure sickle cell and thalassemia – new trial launches
NCT ID NCT06647979
First seen Jan 06, 2026 · Last updated May 23, 2026 · Updated 29 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. Up to 10 people aged 13-40 with severe forms of these blood disorders will receive their own edited stem cells after a short course of chemotherapy. The goal is to see if this one-time treatment can safely and permanently cure the disease without the need for long-term medication.
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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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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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