First-Ever gene therapy targets brain cells in devastating childhood disease
NCT ID NCT04833907
Summary
This study is testing a new, one-time gene therapy for children with Canavan disease, a severe and fatal genetic brain disorder. The therapy is delivered directly into the brain during a surgical procedure, aiming to replace the faulty gene in the specific brain cells that cause the disease. Researchers will check if the treatment is safe and if it can improve brain health and motor skills in up to 24 children.
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Contacts and locations
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Locations
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Dayton Children's Hospital
Dayton, Ohio, 45404, United States
Conditions
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