Gene-Editing breakthrough aims to stop sickle cell pain
NCT ID NCT04774536
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a one-time treatment using CRISPR to fix the sickle cell gene in a patient's own blood stem cells. Up to 9 people aged 12 to 35 with severe sickle cell disease will receive the edited cells. The goal is to reduce painful blockages in blood vessels and improve quality of life, though long-term medication may still be needed.
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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
Contact Email: •••••@•••••