CRISPR gene editing offers hope for sickle cell patients
NCT ID NCT04774536
First seen Apr 20, 2026 · Last updated May 19, 2026 · Updated 7 times
Summary
This early-stage trial tests a one-time infusion of the patient's own blood stem cells, which have been corrected using CRISPR gene editing to fix the sickle cell mutation. The goal is to reduce painful blockages in blood vessels (vaso-occlusive events) and improve quality of life. The study enrolls 9 people aged 12 to 35 with severe sickle cell disease and will monitor safety and effectiveness over time.
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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
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Contact
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Locations
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UCSF Benioff Children's Hospital
RECRUITINGOakland, California, 94609, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of California, Los Angeles
RECRUITINGLos Angeles, California, 90095, United States
Contact Email: •••••@•••••
Contact
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Conditions
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