One-Shot gene therapy tested to halt fatal childhood brain disease
NCT ID NCT02725580
Summary
This study tested a one-time gene therapy injection for children with a rare, fatal genetic brain disorder called CLN6 Batten disease. The therapy aimed to deliver a working copy of the faulty CLN6 gene directly to the nervous system to slow or stop disease progression. Researchers monitored 13 children for two years to check the treatment's safety and see if it helped preserve their motor skills, language, and cognitive function.
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Contacts and locations
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Locations
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Nationwide Children's Hosptial
Columbus, Ohio, 43205, United States
Conditions
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