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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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.
Contacts and locations
Locations
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UCSF Benioff Children's Hospital
RECRUITINGOakland, California, 94609, United States
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University of California, Los Angeles
RECRUITINGLos Angeles, California, 90095, United States
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