First human trial aims to cure sickle cell with One-Time gene therapy
NCT ID NCT04819841
Summary
This early-stage trial is testing a new gene therapy called nula-cel for people with severe sickle cell disease. The treatment involves collecting a patient's own blood stem cells, modifying them in a lab to produce healthy hemoglobin, and then returning them to the patient. The main goals are to check if the treatment is safe and to see if it can reduce or eliminate sickle cell symptoms long-term.
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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Contacts and locations
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Study contacts
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Contact
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Locations
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Children's Hospital Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Lucile Packard Children's Hospital
RECRUITINGPalo Alto, California, 94304, United States
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
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Washington University
RECRUITINGSt Louis, Missouri, 63110, United States
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Conditions
Explore the condition pages connected to this study.